Commitment to conduct a limited pharmacokinetic study in patients to characterize the absorption and elimination phases of the 100 mg/mL formulation and to compare the 50 mg/mL versus 100 mg/mL preparations (via crossover).
Single and multiple-dose pharmacokinetics (PK), pharmacodynamics (PD) and safety study in pediatric patients aged 12 to 16 years. Protocol submission by: December 15, 2004 (6 mos. post-approval) Study start: July 15, 2005 (1 year post-approval)
Sponsor has been issued a PREA non-compliance letter on 7/17/13.
Zegerid® (Omeprazole and Sodium Bicarbonate)
Conduct a study of Entereg for the acceleration of gastrointestinal recovery in pediatric patients age 0 to 1 month undergoing bowel resection surgery. The study will measure population pharmacokinetic parameters, safety, and time to first tole
Per FDA letter dated 05/11/2016, this PMR has been released.
A deferred pediatric study under PREA for the treatment of schizophrenia in pediatric patients ages 13 to 17. A study of the efficacy and safety of iloperidone tablets in the relevant pediatric population.
On 9/24/13 the sponsor requested for deferral extension for PREA PMRs 4-1 and 4-2 and the Agency granted the request on 11/4/13.
To perform Segment III (peri- and post-natal development) studies, as per the ICH M3, S5A, S5B, and S5B(M) Guidances to Industry..
Original milestone: January 1, 2009. Sponsor is awaiting further FDA guidance before proceeding.
Deferred pediatric study under PREA for the treatment of relief of mild to moderate acute pain in pediatric patients ages 12 to 17 years.
Study complete, Report submitted 2/12/15 as S-009, UF not paid
The applicant currently proposes to develop a liquid pediatric formulation and then perform a pharmacokinetic (PK) study in adults comparing the pediatric formulation to an approved pediatric ibuprofen product. The proposed PK study is a single
The Sponsor submitted the study report, but they did not submit the NDA to support the pediatric dosage form because they do not plan on ever marketing the pediatric product.
Deferred pediatric studies under PREA for the treatment of chronic hepatitis B virus infection in pediatric patients ages 2 to <12 years of age.
2nd Deferral Extension granting letter issued on 10/27/2016. The final report submission due date revised from 12/31/16 to 06/30/2018
Conduct a labeling comprehension study to ensure that patients are able to read and use the device in the manner specified in them labeling.
Study completed, results to be submitted
Deferred pediatric study under PREA for treating heartburn associated with non-erosive GERD in pediatric patients aged 1 year to 11 years.
The study has not begun but does not meet the criterion for delayed. Deferral extension granted until 12/31/2018
You have committed to the conduct of a clinical trial designed to assess the potential effects of apomorphine on the QTc interval. Due to the complexity of the design of this study, submit the protocol for review and discussion prior to study i
The study is delayed. Currently negotiating the study protocol for the parent study with the FDA. An Acknowledge Revised Milestones letter was sent on June 2, 2015.
Studies to determine dosing for neonates to 2 years of age (based on human extrapolation and/or animal models). i. Protocol submission: Within 6 months of the date of this letter ii. Study start: Within 6 months of agreement to the protocol iii
Supplement submitted to fulfill the PMR January 31, 2007;submission assigned (NDA#022176), submission did not satisfy the PMR as noted in the FDA NA letter dated March 8, 2008 and the PMR Not Fulfilled letter dated August 9, 2013.
Deferred pediatric study under PREA for the treatment of candidemia and Candida infections in pediatric patients ages 0 to 3 months.
The Sponsor met the goal of submitting the final protocol. The study has been initiated.
Deferred pediatric study under PREA for the maintenance of remission in Crohn?s disease in pediatric patients ages zero to 17 years of age.
The final study report was submitted to FDA on 6/30/2015
You are required to assess the safety and effectiveness of EQUETRO as a treatment for acute manic and mixed episodes associated with Bipolar I Disorder in pediatric patients ages 10 to 17 (children and adolescents). Due to extensive prior exper
The study/trial has not begun but does not meet the criterion for delayed.Original Final Report Due Date: 1/30/12 Deferral Extension Granted per FDA letter dated 7/10/13 revising the Final Report Submission date to 12/29/17.
Deferred pediatric study under PREA for the maintenance of remission of ulcerative colitis in pediatric patients ages 5 to 17 years to evaluate safety, effectiveness, and pharmacokinetics with Apriso (mesalamine granules) in at least two dosing
The final report milestone was missed. Deferral Extension Requested 7/23/2013. Denied per FDA letter dated 9/9/2013.
Deferred pediatric study under PREA for the management of pain in patients where an opioid analgesic is appropriate in pediatric patients ages 0 to 16 years.
Deferral Extension Requested 03/01/2016.  Denied per FDA letter dated 04/15/2016.
Dilaudid® and Dilaudid® HP (Hydromorphone Hydrochloride)
Deferred pediatric study under PREA for the treatment of relief of mild to moderate acute pain in pediatric patients ages 2 to 12 years.
Deferral Extension Requested 03/23/2016.  Denied per FDA letter dated 05/02/2016.
A pediatric study in patients aged 6 - 17 years to obtain pharmacokinetic data pertinent to pediatric dosing of tolvaptan.
The final Protocol was submitted on 05 Nov 2013. Subject enrollment has not initiated. The trial is planned to enroll up to 100 subjects.
Single and multiple-dose pharmacokinetics (PK), pharmacodynamics (PD) and safety study in pediatric patients aged 2 to 11 years. Protocol submission by: December 15, 2004 (6 mos. post-approval) Study start: July 15, 2005 (1 year post-approval)
Deferred pediatric study under PREA for the treatment of hypertension in pediatric patients ages 6 to 16 years.
CSR withdrawn; applicant granted deferral extension for CMC prep of pediatric formulation to satisfy Written Request
Human pharmacokinetic study in adult subjects to compare and evaluate the absorption, distribution and elimination of Ca and Zn-DTPA via inhalation using a commonly available jet type nebulizer (FDA approved model to be selected by the sponsor)
Per AP Letter Aug 11, 2004, the protocol due was date Feb 11, 2005, 6 month after application approval. Draft Adult PK protocol submitted August 10, 2006 Confirming status of protocol review with PK and clinical team Products is for treatment o
Deferred pediatric study under PREA for the treatment of ulcerative colitis in pediatric patients of all ages.
Original Final Report Due Date: 12/31/2010; Deferral Extension granted per FDA letter dated 03/25/2013. Protocol has been submitted
Deferred pediatric study under PREA for the treatment of HIV in pediatric subjects from 2 to 18 years of age. This study will determine the maraviroc exposure (pharmacokinetics profile) followed by 48 weeks of dosing, with efficacy based on vir
Per FDA letter dated 11/09/2016, this PMR has been fulfilled.
To conduct a post-marketing multi-center, randomized, placebo-controlled, large, simple safety study to evaluate the effects of long term use of Perforomist Inhalation Solution in patients with COPD. The objective of this trial would be to dete
Deferred pediatric study under PREA for the prevention of acute nausea and vomiting associated with moderately emetogenic cancer chemotherapy in pediatric patients ages 29 days to 16 years.
Per FDA letter dated 05/24/2016, this PMR has been released.
Completion of a controlled clinical study to verify and describe the clinical benefit of clofarabine in pediatric ALL. Your proposed Phase 3 study to be possibly conducted by the COG does not appear to have a realistic chance of showing a clini
Revised milestone date for protocol is Sept 2012; study completion is June 2019 and study report is December 2019. The clofarabine arm of the study was closed based on results of pre-planned safety analysis. The Applicant will be discussing a n
Deferred pediatric study under PREA for the treatment of Irritable Bowel Syndrome with constipation in pediatric patients ages 6 to 17. The design consists of a 12-month multi-center, double-blinded, placebo-controlled safety and efficacy study
Per FDA letter dated 09/01/2016, this PMR has been released.
Conduct a descriptive study of the use of Coartem Tablets in non-immune travelers. For a period of five years following approval, collect baseline patient demographic information (including age, weight, height, sex, race, prior medications and
The final report was due in April, 2016.
A deferred pediatric study under PREA for the prevention of nausea and vomiting in patients receiving moderately and/or highly emetogenic chemotherapy for up to 5 consecutive days in pediatric patients ages 2 to 17. A study of the efficacy and
Deferral Extension Requested 08/23/2016.  Denied per FDA letter dated 10/03/2016.
Commits to performing an HPA axis suppression study in no less than 60 evaluable subjects (30 adults and 30 adolescents 12 to 17 years of age) using cosyntropin stimulation testing conducted as labeled with pre- and exactly-30-minutes-post-stim
Establish a registry for children aged 2 to < 6 years to enroll approximately 200 patients and follow them for 5 years. Data collection will be at least monthly for renal function and blood pressure and yearly for growth and development. Submit
Forteo User Registry Study (B3D-MC-GHBX[2]) Study B3D-MC-GHBX(2) is a prospective cohort study that will allow voluntary registration of adult Forteo (teriparatide, rPTH[1-34]) users in the United States during a 5 year enrollment period that w
Commitment to determine whether Quadramet dose adjustment is needed in patients with renal insufficiency since Quadramet is primarily excreted intact in the urine.
Lantheus report based on previous information from the previous owners the likelihood of receiving IRB approval to conduct this type of study would be difficult. Lantheus is currently conducting a thorough review of the published literature
Conduct a non-interventional, prospective, observational study to provide additional safety data on important clinical events. The duration of the study will be 5 years from initiation of the study; data will be reviewed on an interim basis eve
Submit the results of the proposed phase III trial (AALL0434) to be conducted by the Children's Oncology Group to demonstrate nelarabine's clinical benefit.
Original 3-year follow-up due 4Q 2015 with final report due 2016. Three year follow-up is delayed. Applicant projects 3-year follow-up to be completed 3Q017 and Final report completion by 2Q2018. (Revised milestones acknowledged Dec 31,2014.)
A deferred pediatric study under PREA for the prevention of nausea and vomiting in patients receiving moderately and/or highly emetogenic chemotherapy for up to 5 consecutive days in pediatric patients ages 2 to 17. A study to examine the pharm
Original Final Report Due Date: 10/31/2014; Deferral Extension granted per FDA letter dated 10/03/2016.
Continuation and modification of your ten-year epidemiological study assessing whether treatment with pioglitazone hydrochloride is associated with an increased risk of bladder cancer in men and women with diabetes.
ActoPlus Met® (Pioglitazone and Metformin)
Duetact® (Glimepiride and Pioglitazone Hydrochloride)
Deferred pediatric study under PREA for the treatment of type 2 diabetes in pediatric patients ages 11 to 16, inclusive.
Original Final Report Due Date: 12/31/2010. Per FDA letter dated 04/29/2013, Final Report due date extended to 04/25/2018. Second Deferral Extension Granted per FDA letter dated 01/25/2016.
A prospective, randomized, active-controlled trial comparing the risk of developing acute kidney injury in patients undergoing bowel cleansing using Visicol or OsmoPrep as compared to patients undergoing bowel cleansing using polyethylene glyco
Visicol® (Sodium Phosphate Monobasic Monohydrate & Sodium Phosphate Dibasic Anhydrous)
To evaluate the potential for a serious risk of carcinogenicity, conduct a long-term (2 year) rodent carcinogenicity study in the rat. Submit the carcinogenicity protocol for a Special Protocol Assessment prior to initiating the study.
Additional rat toxicity study was required prior to initiating carcinogenicity study. FSR to be submitted during next reporting period (sponsor sent 12-JUL-16)
An open-label pharmacokinetic and safety study or studies of an ageappropriate formulation of Zorvolex in pediatric patients 6 years to less than 17 years of age with acute pain.
An open-label pharmacokinetic and safety study or studies of an ageappropriate formulation of Zorvolex in pediatric patients 2 years to less than 6 years of age with acute pain.
A pharmacokinetic, safety, and efficacy study or studies of an age-appropriate formulation of Zorvolex in pediatric patients 1 year to less than 2 years of age with acute pain.
Commitment to perform a long-term safety and efficacy trial in panic disorder patients to address the questions of maintenance efficacy and of the risks of long-term use. Applicant has agreed to initiate the trials within six months of final ag
This PMC was to provide long-term safety and efficacy data in panic disorder patients. The sponsor has not initiated the study and submitted a waiver request dated 8/10/11. DPP denied the request, issued a PMC Not Fulfilled letter on 1/13/12, a
To conduct a minimal in vitro genetic toxicology screen (one in vitro mutagenicity assay and one in vitro assay for chromosome damage) to characterize the toxicological safety of the drug product shelf life specifications (stability specificati
Original milestone: October 1, 2007. Agency letter dated 2/24/2009 informed Sponsor that PMR was not adequately completed. Sponsor reports that they have reinitiated the study as of Jan 2014.
Deferred pediatric study under PREA for the treatment of Stage 3 and Stage 4 chronic kidney disease in pediatric patients ages twelve to sixteen years.
Per FDA letter dated 10/18/2016, this PMR has been fulfilled.
Randomized, double-blind, dose-controlled clinical trial of fospropofol disodium injection in adolescent patients (12 through 18 years old) undergoing upper endoscopy. Pharmacokinetics will be studied using a population PK approach.
NDA WD 06/28/2012. Applicant does not plan to complete any of the studies that were initiated.
Explore potential agonist (direct or indirect) effects on adrenergic tissue.
To conduct pharmacokinetics studies in neonates and children under 2 years of age who do not have mature renal function.
Conduct a study of Entereg for the acceleration of gastrointestinal recovery in pediatric patients age greater than 1 month up to 16 years undergoing bowel resection surgery. The study will measure the time to first tolerated feed, population p
To conduct pharmacokinetic studies to determine what dose adjustments may be needed in patients with renal and liver impairment.
NDA Withdrawn withdrawal requested April 29, 2016 Guerbet indicates study files acquired during the acquisition of the product are deficient and data not usable.
Applicant Holder commits to conduct one or more studies in pediatric patients to address among other things, dosage, administration, instrumentation or other technical features which may affect performance and safety.  Also to evaluate OPTISON
Deferred pediatric study under PREA for the treatment of mild to moderate active Crohn's disease involving the ileum and/or the ascending colon in pediatric patients ages 5 to 17 years.
Adult clinical study to address longer-term effectiveness of carbamazepine in Bipolar I Disorder.
No new information for this reporting period. Previous reporting period reported the following: The study/trial has not begun, and the Final Report Submission date of 6/30/12 has passed. Validus is diligently making every effort to start this s
Complete study of long-term follow-up (3 years) in 150 patients with myelodysplastic syndromes (MDS) receiving Exjade to evaluate safety (including cardiac, hepatic, endocrine and renal) and hematologic and clinical benefit of Exjade in these p
Original Study report due December 31, 2009. Applicant had difficulty with enrollment and modified the protocol to try to resolve the problem. Nov 4, 2013, Applicant requested release from PMR. May 28, 2013, FDA responded that Study for PMR 199
To study the pharmacokinetics, efficacy and safety of tramadol hydrochloride extended-release capsules for the management of moderate to moderately severe chronic pain in pediatric patients ages ¡Ý 2 to 17 years.
Sponsor is awaiting for the Agency’s response for the clarification questions submitted
A large randomized, double-blind, active- and placebo-controlled trial to compare the risk of clinically significant neuropsychiatric events, including but not limited to suicidality, in individuals using Chantix (varenicline), bupropion, nicot
Clarify the possible effects on developing fetal adrenergic tissue.
A pregnancy registry must be established to evaluate the safety of this product in pregnant women and their offspring. You will be required to evaluate the safety of VIBATIV (telavancin) use during pregnancy by developing and maintaining a pros
To conduct a clinical trial in pediatric patients aged 2 to < 18 years who have iron deficiency anemia and who are receiving either hemodialysis or peritoneal dialysis. In addition to any other items, the trial will obtain pharmacokinetic (PK),
Deferred Study completion was due March 2016. Final report is due March 2017. Studies were closed out and a new study initiated.
An observational study of the association of azithromycin use with cardiovascular death, compared to amoxicillin or other antibacterial drugs, in one or more study population(s) with sufficient representation of cardiovascular disease and cardi
To conduct a clinical trial, per FDA Guidance [Drug Interaction Studies-Study Design, Data Analysis, Implications for Dosing, and Labeling Recommendations], in order to determine the effects of a strong CYP1A2 inhibitor such as fluvoxamine on t
Collect and analyze data regarding pregnancy outcomes for 10 years after approval of Impavido (miltefosine) in women who become pregnant while taking Impavido (miltefosine) or during 5 months after end of Impavido (miltefosine) therapy
Assessment of efficacy and safety of OraVerse in patients from 2-6 years of age. This study should be a randomized, sham-injection controlled, double-blinded study comparing the times to return of normal sensation and normal function following
Per FDA letter dated 03/17/2016, this PMR has been released.
A commitment to conduct a well-controlled, prospective growth study of the effects of Tri-Nasal Spray on growth in Prepubertal children.  The results of this study should be reported to the Agency by January 24, 2003.  Applicant will consult th
To adequately assess photosafety to support the drug as described in the 2003 Guidance for Industry: Photosafety Testing.
Original milestone: March 1, 2008 Sponsor is awaiting further FDA guidance before proceeding.
A safety study in 100 pediatric patients, ages 10-17 years, of single dose cefazolin preoperative prophylaxis using the dose equivalent to 2 gram adult exposure (as determined in the PK study) in pediatric patients.
The sponsor commits to conduct a Phase IV confirmatory clinical study to determine the effect of Luveris on time to clinical pregnancy. The following is the timeline for this commitment: Protocol submission: Accomplished in the October 5, 2004
A single-dose pharmacokinetic and pharmacodynamics trial of empagliflozin in pediatric patients 10 to 17 years (inclusive) with type 2 diabetes mellitus.
The Final Report was submitted to FDA on 10/12/2016.
Synjardy® (Empagliflozin and Metformin Hydrochloride)
Conduct a Long term safety study to further support the administration of Avinza and the safety profile of fumaric acid in high dose patients. The study will be conducted in approximately 100 patients receiving Avinza doses in excess of 500 mg
A single dose, pharmacokinetic, open-label, clinical study in healthy lactating women. Concentrations of pregabalin will be assessed in maternal plasma and breast milk so as to estimate potential infant exposure.
Conduct a clinical study to evaluate recurrence or partial response to treatment in patients treated with Metvixia/Aktilite PDT.
Complete recruitment was to be completed by 12/1/2009.
Deferred pediatric study under PREA for the management of fibromyalgia in pediatric patients ages 13 to 17.
Enrollment is ongoing. As of 07 March 2016, 169 patients have been enrolled.
Deferred pediatric study under PREA for the treatment of partial onset seizures in pediatric patients ages 1 month [44 weeks gestational age] to 16 years.
Pediatric studies – PK and safety. Milestones dates were revised to September 30, 2018.
To provide literature data or perform new studies to describe the safety of Ioxilan in venographic procedures.
Deferred pediatric study under PREA for cleansing of the colon as a preparation for colonoscopy in pediatric patients ages birth to 16 years.
Sponsor has withdrawn this NDA and does not intend to conduct this study
HalfLytely® and Bisacodyl Tablet (PEG-3350, Sodium Chloride, Sodium Bicarbonate, and Potassium Chloride; and Bisacodyl)
Deferred pediatric studies under PREA for the treatment of cUTI in pediatric patients ages 0-18 years.
Deferred pediatric study under PREA for the treatment of Major Depressive Disorder in pediatric patients ages 7 to 17.
Valeant anticipates initiating study V01-BUPA-401 in Q4 2016 to meet NDA 22-108 Postmarketing Requirement (PMR 133-1) established under the Pediatric Research Equity Act (PREA) for conducting pediatric studies in the major depressive disorder i
VALEANT PHARMACEUTICALS NORTH AMERICA LLC
Deferred controlled effectiveness study under PREA for the acute treatment of migraine attacks with or without aura in pediatric patients ages 12 years to 17 years.
The final study report deadline has passed. The sponsor is currently negotiating the final study protocol with the FDA.
To provide information or conduct pharmacokinetics studies in children 2-16 years of age.
Completion of the clinical confirmatory trial required by the Accelerated Approval regulation, as outlined in your submission dated May 13, 1998, and revised as agreed to in your facsimile of May 28, 1998.
The Sponsor has not started the study yet. There is ongoing discussion with the Division regarding the study design.
Longitudinal studies involving follow up on case report forms and placement of data into a database for periodic analyses to determine length of treatment, safety profile, and other factors related to drug effectiveness. a. Protocol submission:
To conduct a clinical trial in pediatric patients aged 2 to < 18 years who have iron deficiency anemia and chronic kidney disease that does not require dialysis. In addition to any other items, the trial will obtain pharmacokinetic (PK), pharma
Deferred trial completion was due March 2016. Final report is due March 2017. Studies were closed out and a new study initiated
Deferred pediatric study under PREA for the treatment of for short-term treatment (7 to 10 days) of gastroesophageal reflux disease (GERD), as an alternative to oral therapy in patients who are unable to take Protonix (pantoprazole sodium) Dela
The final report milestone was missed. Deferral Extension Requested 1/4/13. Denied per FDA letter dated 5/30/13.
Deferred pediatric study under PREA for the treatment of the control of serum phosphorus in patients with Chronic Kidney Disease (CKD) on dialysis in pediatric patients ages < 1 month to 16 years old.
Per FDA letter dated 12/14/2016, this PMR has been fulfilled.
To conduct a toxicology study of adequate dose and duration to characterize the toxicological safety of the drug product shelf life specifications (stability specifications).
Deferred pediatric study under PREA for the treatment of cSSSI in pediatric patients ages 0 to 17 years.
Deferred pediatric study under PREA for the treatment of GERD in pediatric patients ages 0 to 16.
Submit an analysis of overall survival after 10 years of follow-up for IES.
Conduct a trial to determine the efficacy and safety of the use of deferiprone to treat iron overload in patients with sickle cell disease and transfusional hemosiderosis who have not been adequately treated with available chelating agents. Sub
Recruitment is slow, but the FDA requested enrollment continued to obtain more clinical data. Revised milestones acknowledged Nov 14, 2014 to complete study 2/1017 and submit the final study report July 2017
Establish a registry in order to perform an enhanced pharmacovigilance study of agranulocytosis. Submit a protocol to establish the registry and describe procedures for this enhanced pharmacovigilance prior to commencing the study. Procedures s
You will submit a protocol for a prospective study of the effect of buprenorphine on the liver, using a methadone-treated control group. The study should be sufficiently large and of sufficient duration to determine whether buprenorphine causes
To conduct a rodent carcinogenicity study in the mouse. Prior to initiating the study, you will submit and receive agreement on the carcinogenicity protocol under a Special Protocol Assessment (SPA).
Conduct and submit the results of at least one multicenter, randomized clinical trial establishing the superiority of ceritinib over standard therapy in adult patients with ALK-rearranged (ALK-positive) metastatic NSCLC who have been previously
Conduct a clinical trial to evaluate the systemic exposure and safety of 450 mg Zykadia (ceritinib) taken with a meal and 600 mg Zykadia (ceritinib) taken with a light meal as compared with that of 750 mg Zykadia (ceritinib) taken in the fasted
To conduct a long-term rodent carcinogenicity study in the rat. Prior to initiating the study, you will submit and receive agreement on the carcinogenicity protocol under a Special Protocol Assessment.
To conduct a Pregnancy Pharmacovigilance Study to collect pregnancy registry data to evaluate pregnancy outcomes and infant outcomes following exposure to vismodegib. This study will include a mechanism to collect, classify, and analyze data on
To conduct a large, simple, randomized, placebo controlled trial of rasagiline added to standard therapy in approximately 5000 Parkinson's disease patients for a duration of 36 months to assess the relative risk of melanoma.
The pilot study that will guide the development of the final protocol for the main study is ongoing.
To study the pharmacokinetics, efficacy and safety of Ryzolt for the the management of moderate to moderately severe chronic pain in pediatric patients ages less than or equal to 2 to 17 years.
The study has not been initiated, but does not meet the criterion for delayed.
Deferred pediatric study under PREA for treating heartburn associated with non-erosive GERD in pediatric patients aged 12 years to 17 years.
Per FDA letter dated 07/08/2016, this PMR has been fulfilled.
Deferred pediatric studies under PREA for the treatment of traveler's diarrhea in pediatric patients ages 3 years to 12 years. Final Report Submission: May 1, 2009.
Sponsor requested a deferral extension. Deferral extension granted 7/9/13
Deferred pediatric studies under PREA for the treatment of cIAI in pediatric patients ages 0-18 years.
Provide a rationale to support the safe use of the novel excipient cocoyl caprylocaprate that would preclude the submission of preclinical studies.
Sponsor sumitted studies to support their argument that PMC 2a and 2b were unnecessary. However the FDA did not agree and a letter issued to sponsor on 12/28/09.  Novartis submitted new timelines on 4/13/10.
(2a) If the proposed rationale submitted under Postmarketing commitment number 2 is determined to be inadequate, the following study will be required: A dermal carcinogenicity evaluation of cocoyl caprylocaprate in two species. One of these stu
To cooperate with U.S.-based public health agencies in evaluating data on the use of LEVAQUIN (levofloxacin) in a large U.S population for inhalational anthrax (post exposure) prophylaxis, should an exposure occur. This includes long-term safet
Deferred pediatric study under PREA for the treatment of migraine in pediatric patients ages 12 - 17 years.
The Sponsor submitted a request for withdrawal of supplement S-005. FDA acknowledged withdrawal of this supplement on 1/11/13. FDA will release PMR 298 following publication of the FR notice.
A 10 year, observational study to prospectively evaluate the incidence of fibrosing colonopathy in patients with cystic fibrosis treated with Creon (pancrelipase) Delayed- Release Capsules in the US and to assess potential risk factors for the
A 10 year, observational study to prospectively evaluate the risk of transmission of selected porcine viruses in patients taking Creon (pancrelipase) Delayed-Release Capsules.
Deferred safety and pharmacokinetic pediatric study under PREA in pediatric patients with migraine with or without aura ages 6 years to 11 years. Upon review of additional safety and effectiveness data in pediatric patients ages 12 to 17 years,
The final study protocol deadline has passed, and the sponsor has not yet submitted a study protocol.
Deferred controlled effectiveness study under PREA for the acute treatment of migraine with or without aura in pediatric patients ages 6 years to 11 years. Upon review of additional safety and effectiveness data in pediatric patients ages 12 to
Commits to performing a safety and efficacy study in non-Caucasian subjects, with particular attention to subjects of African-American and Asian ethnicity. Protocol Submission: Within 4 months of receipt of approval letter Study Start: Within 6
A prospective observational cohort study to examine the incidence of pancreatic malignancy and thyroid neoplasm in patients with Type 2 diabetes mellitus initiated on Byetta compared to patients initiated on other antidiabetic agents
Human pharmacokinetic study in pediatric subjects to compare and evaluate the absorption, distribution and elimination of Ca and Zn-DTPA via inhalation using a commonly available jet type nebulizer with the intravenous route after an event in w
Per AP Letter Aug 11, 2004, the protocol due was date Feb 11, 2005, 6 month after application approval. Letter dated April 24, 2006 referenced a due date of Aug 11, 2004, 24 months after application approval date A 3 month extension was request
Deferred pediatric studies under PREA for the treatment of chronic idiopathic constipation in pediatric patients ages 0 to 17 years.
Per FDA letter dated 02/11/2016, this PMR has been released.
We are deferring pediatric studies for ages 6-17 years for this application.
Protocols have been submitted [...]. Pediatric study is ongoing.
Deferred pediatric study under PREA for the treatment of post-operative nausea and vomiting pediatric patients ages 0 to less than 17 years of age.
Original Final Report due date: 12/31/09. Deferral extension granted per FDA letter dated 4/12/13. The final report milestone date extended until 1/31/20.
Deferred pediatric study under PREA for the use of VIREAD treatment, in combination with other antiretroviral agents, of HIV-1 in pediatric patients ages birth to 2 years of age. Due to safety concerns for this age group, we are waiting for com
Deferral extension granting letter issued on 07/02/2013. The trial has not initiated but does not meet the criterion for delayed. The final report due date revised from 01/31/2010 to 12/31/2019.
Sponsor commits to undertake a Phase 4 trial to determine the need for and how to adjust Ferriseltz by body size in pediatric patients.
Deferred pediatric bioavailability study: To Assess the Safety, Tolerability and Pharmacokinetics of Cycloset in 10 to 16 year old Type 2 Diabetic Subjects.
Per FDA letter dated 01/06/2017, this PMR has been fulfilled.
To conduct and submit a final study report for a postmarketing study of thromboembolic events among women prescribed Lybrel compared to women prescribed cyclic oral contraceptives containing 20mcg ethinyl estradiol. The postmarketing study will
Lybrel® (Levonorgestrel and Ethinyl Estradiol)
Deferred safety and pharmacokinetic pediatric study under PREA in pediatric patients with migraine with or without aura ages 12 years to 17 years.
To evaluate efficacy and safety of dalteparin in pediatric cancer patients. Studies using dalteparin for venous thromboembolism (VTE) treatment in all age ranges of the pediatric population should be performed.
Deferral granted December 22, 2015 due to enrollment difficulties. Final report submission due December 31, 2018.
Conduct clinical trials of ropivacaine usage and pharmacokinetics in children and infants.
To develop and maintain a prospective, observational pregnancy exposure registry study conducted in the United States that compares the pregnancy and fetal outcomes of women exposed to Cymbalta during pregnancy to an unexposed control populatio
As of March 2016, 109 prospective cases were enrolled into the current registry with INC Research. No longer able to execute VAMPPS protocol. Continue study  proposal to add a database to the current strategy is being evaluated.
Determine the no observable effect level on electrocardiographic changes in the dog model.
Randomized, double-blind, dose-controlled clinical trial of fospropofol disodium injection in children (ages 3 up to 12 years old) undergoing sedation for magnetic resonance imaging (MRI). Pharmacokinetics will be studied using a population PK
Deferred long-term open label safety study under PREA in pediatric patients with migraine ages 6 years to 11 years. Upon review of additional safety and effectiveness data in pediatric patients ages 12 to 17 years, we will make a determination
A 24-month oral carcinogenicity study of lumacaftor in rats
Evaluate the safety and treatment response (using sustained virologic response as the primary endpoint) of TECHNIVIE (ombitasvir, paritaprevir, and ritonavir) in a cohort of pediatric subjects 3 to less than 18 years of age with chronic genotyp
Enrollment for the study is currently ongoing, 18 of 36 subjects enrolled
Technivie® (Ombitasvir, Paritaprevir, and Ritonavir)
Submit a final report of your analysis of the persistence of treatment-emergent, ombitasvir or paritaprevir resistance-associated substitutions through Post- Treatment Week 48 in ongoing trials of HCV genotype 4 infected subjects.
The preclinical rodent studies found an increased risk for lymphoma and follicular cell thyroid adenoma in the studies evaluating an oral formulation of pimecrolimus. We agree to conduct a registry study of pediatric patients (aged 2-17, with e
A commitment to conduct a registry study of pediatric patients with atopic dermatitis to address the risk of developing cutaneous or systemic malignancies in patients who have long term intermittent treatment with Protopic Ointment 0.03% or 0.0
BioMarin commits to designing and implementing a registry of patients with PKU being treated with Kuvan (sapropterin dihydrochloride) that will be established to obtain longterm clinical status information. Information will be collected on pati
Deferred pediatric studies under PREA for the adjunctive treatment of partial onset seizures in pediatric patients ages 1 month up to 17 years.
Forty-five subjects have been enrolled in a pediatric pharmacokinetic study and 74 subjects have been enrolled in an open-label safety and tolerability extension study. Twenty-three subjects are enrolled in a long-term pediatric efficacy and sa
Randomized, double-blind, dose-controlled clinical trial of fospropofol disodium injection in infants and very young children (ages one month up to three years old) undergoing sedation for procedures such as lumbar puncture and/or MRI. Pharmaco
To conduct a study to assess the immunogenicity of Urokinase after primary dosing. Abbott will agree on a study design with CBER prior to initiating the study.
The Applicant needs to run tests on patient samples to complete the study. The Applicant has not submitted a final report that was due December 2004. The product is not currently being marketed. The associated IND has been inactivated.
Deferred pediatric study under PREA for the treatment of candidemia and other forms of Candida infections (intra-abdominal abscess and peritonitis) in pediatric patients ages zero months to sixteen years of age.
Final report submission date is 11/29/19
Conduct a study to comprehensively address device durability and reliability.
Protocol is being revised for resubmission.
Deferred pediatric study(ies) under PREA for the treatment of overactive bladder in the subgroup of pediatric patients with neurologic disease ages 6 to 16 years, 11 months.
Original Final Report Due Date: 08/31/2015; Deferral Extension granted per FDA letter dated 7/9/2015. The study has enrolled 33 subjects. 32 subjects have completed the open label phase, there was 1 subject that had terminated early. The Sponso
Deferred pediatric study under PREA for the treatment of migraine in pediatric patients ages 12 to 17 years old.
Original Final Report Due Date: 03/31/2007; Deferral Extension granted per FDA letter dated 07/03/2013
A study to bank samples from inflammatory bowel disease patients treated with Simponi (golimumab) for future evaluation to identify genetic mutations and other biomarkers that may predispose them to developing Hepatosplenic T-Cell Lymphoma (HST
Develop and maintain a prospective, observational pregnancy exposure registry study conducted in the United States that compares the pregnancy and fetal outcomes of women exposed to Savella (milnacipran HCl) during pregnancy to an unexposed con
Deferred pediatric study under PREA for the treatment of Gastroesophageal Reflux Disease (GERD): Healing of Erosive Esophagitis, Maintenance of Healing of Erosive Esophagitis, Symptomatic Gastroesophageal Reflux Disease in pediatric patients ag
AstraZeneca submitted a proposal on June 22, 2011. The FDA responded on October 1, 2014. No study has been initiated for the maintenance of healing of healing of erosive esophagitis portion of this PMR in patients aged 1 year to 11 years.
Deferred pediatric study under PREA for the topical treatment of moderate to severe forms of scalp psoriasis in pediatric patients ages 12 to 17. Final Report Submission: April, 2006
Final study report due on April 30, 2006, but has not been submitted to date. A PREA waiver request was submitted on October 11, 2004, but a determination has not been made.
To provide a ribavirin pregnancy registry post-marketing surveillance study. Protocol Submission: Within 2.5 months of the date of this post-approval commitment letter. Follow-up teleconference: Within three months of the date of this post-appr
Deferred pediatric study under PREA for the treatment of moderate to moderately severe pain in pediatric patients.
Per FDA letter dated 01/17/2017, this PMR has been released.
Conduct a systemic bioavailability study using a properly validated assay method to measure methyl aminolevulinate and metabolite (aminolevulinic acid) in plasma under the labeled condition of use in subjects with multiple (8-10) actinic kerato
Study start date of 1/2/2009 was missed.
Teriparatide Post-Approval Osteosarcoma Surveillance Study (B3D-MC-GHBX). Study B3D-MC-GHBX is an ongoing, case-series study to identify cases of osteosarcoma among men and women 40 years of age and older and determine which cases, if any, have
A clinical trial to evaluate the effect of Kapidex (dexlansoprazole) Delayed Release Capsules on bone homeostasis. The primary endpoint will be biomarkers of bone formation and bone resorption. Treatments will include: placebo, dexlansoprazole
Deferred pediatric trial under PREA: A randomized, controlled, parallel group superiority trial in pediatric patients ages 10 through 17 years to evaluate the pharmacokinetics of dimethyl fumarate, and the safety and efficacy of dimethyl fumara
Deferred pediatric studies under PREA in positron emission tomography (PET) imaging in patients with coronary artery disease and left ventricular dysfunction, when used together with myocardial perfusion imaging, for the identification of left
PMR Release pending publication of WD in the FR
Identify dosing adjustments needed in patients with renal insufficiency.
Further explore the specific effects of medication which could interfere with the uptake of Iobenguane Sulfate I 131. This should include a determination of when the medication should be stopped prior to Iobenguane Sulfate I 131 administration
Gilead agrees to conduct a study examining the effects of LETAIRIS on 6-minute walk distance at peak and trough plasma concentrations, and further agrees to reach agreement on an appropriate study design with the Division.
Deferred pediatric study under PREA for the prophylaxis of invasive Aspergillis and Candida infections in patients, who are at risk of developing these infections in pediatric patients two years to twelve years of age.
Per FDA letter dated 10/17/2016, this PMR has been released.
To submit the final trial report for Study A8081007 that includes the final analysis of overall survival as specified in the version of Study A8081007 submitted on February 5, 2013, in order to characterize the effects of Xalkori (crizotinib) t
Deferred pediatric study under PREA to determine the safety of Zemplar (paricalcitol) for the treatment of secondary hyperparathyroidism associated with chronic kidney disease (CKD) Stage 5 in pediatric patients ages 10 to 16 years receiving pe
Complete an adequate and well-controlled clinical study or studies to better assess the ophthalmologic effects of pregabalin.
Opthalmologic Study -Enrollment in this study has been significantly below the original estimates. The Sponsor continues to proceed with the study and is looking at additional options to address slow enrollment and possible alternatives to addr
Develop an educational outreach program for health care providers and patients, focusing on Yasmin's contraindications in patients with renal or hepatic impairment and inpatients predisposed to hyperkalemia. Protocol Submission: Within 3 months
Yasmin® (Drospirenone and Ethinyl Estradiol)
Firm commits to obtain pharmacokinetic data relevant to children with juvenile rheumatoid arthritis, a post-marketing pharmacokinetic study will be conducted in a suitable number of pediatric patients with juvenile rheumatoid arthritis; or in l
To assess the effect of the implant on the corneal endothelium, you will complete a case-controlled study using a subset of approximately 100 patients from Clinical Studies BLP 415-001 and BLP 415-004 who have been implanted with Retisert for a
To assess the effect of the implant on the corneal endothelium, Bausch & Lomb will complete a casecontrolled study using a subset of approximately 100 patients from Clinical Studies BLP 415-001 and BLP 415-004 who have been implanted with Retis
Document the presence or absence of Iobenguane Sulfate I 131 in the breast milk and whether or not it crosses the placenta.
Submit the comparative results for the sensitivity and specificity of the CT/MRI scans in the neuroblastoma patients.
Conduct a double-blinded clinical trial testing a single treatment arm of Amitiza versus placebo in male and female patients with Irritable Bowel Syndrome with constipation, utilizing a higher dose than recommended for this indication (e.g., 16
A deferred pediatric study under PREA for the treatment of schizophrenia in pediatric patients ages 13 to 17. A study to obtain pharmacokinetic data and provide information pertinent to dosing of iloperidone tablets in the relevant pediatric po
Per FDA letter dated 08/24/2016, this PMR has been fulfilled.
Conduct and submit a final report for a non-randomized, controlled, observational study to provide additional safety data regarding the incidence of mortality, liver failure, malignancy, myocardial ischemia or infarction, and rhabdomyolysis, as
The revised milestone proposal submitted on 06/05/15 and was acknowledged on 08/20/2015.
Commitment to continue to collect comprehensive safety data in all ongoing trials. This should include vital signs, electrocardiograms and laboratory data.  The case report forms for these purposes should be reviewed by the division prior to th
Submit surveilance reports to evaluate the potential development of resistance to Coartem Tablets. For a period of five years following approval, submit a yearly report describing the reported resistance to a combination ofartemether and lumefa
A randomized, controlled trial of adequate size and duration to determine whether the use of Uloric is associated with a moderate increase in the risk of serious adverse cardiovascular outcomes as compared to allopurinol.
11/25/15, Semi-annual report submitted, 5708 enrolled.
A study to assess the effectiveness and safety of tolvaptan in the treatment of clinically significant hypervolemic and euvolemic hyponatremia in pediatric patients aged 6 to 17.
Conduct an ophthalmologic study in patients receiving Exjade. Examinations should include distance visual acuity, applanation tonometry, lens photography, and wide angle fundus photography of retina and optic nerve and should be done at baselin
The initial study submitted November 28, 2012 did not fulfill the PMC. (Not fulfilled letter sent March 24, 2015). The Applicant is developing a new protocol to address the PMC. The protocol is under discussion with the Agency.
A randomized, double-blind, placebo-controlled trial to evaluate the effect of long-term treatment with Belviq on the incidence of major adverse cardiovascular events (non-fatal myocardial infarction, non-fatal stroke, and cardiovascular death)
Open label, multicenter single ascending dose study to evaluate pharmacokinetics, safety and tolerability of mirabegron modified release microgranule-based suspension in children from 5 to less than 18 years of age with neurogenic detrusor over
Open label, baseline-controlled, multicenter, sequential dose titration study followed by a fixed dose observation period to evaluate pharmacokinetics, safety and efficacy of mirabegron modified release microgranule-based suspension in children
The study/trial has not begun but does not meet the criterion for delayed.
A long-term observational study using electronic healthcare databases with appropriate linkages conducted in United States and European databases to evaluate the incidence of serious cardiovascular outcomes (both individual and composite outcom
A long-term observational study in electronic healthcare databases with appropriate linkages to prospectively evaluate the incidence of new malignant events (excluding non-melanoma skin cancer) in patients using Myrbetriq (mirabegron).
Deferred pediatric study under PREA for the treatment of short-term management of acute post-operative pain in patients requiring opioid analgesia during hospitalization in pediatric patients ages 6 to 16 years of age.
Protocol submitted, final report due March 2017. It is a two part study, part 1 is ongoing, 2 is pending.
Deferred pediatric study under PREA for the treatment of chronic idiopathic constipation in pediatric patients ages 0 to 18 years. Final Report Submission: January 2, 2008
The study is terminated since the drug is no longer marketed.
A rodent carcinogenicity study in the mouse designed according to "FDA Guidance for Industry-Carcinogenicity Study Protocol Submissions". Submit the carcinogenicity protocol for Special Protocol Assessment prior to initiating the study.
Deferred pediatric study under PREA for the treatment of community acquired pneumonia and pelvic inflammatory disease in pediatric patients.
Per FDA letter dated 01/09/2017, this PMR has been released.
Deferred long-term open label safety study in pediatric patients with migraine with or without aura ages 12 years to 17 years.
The final study report deadline has passed. This study is dependent on completion of PMR 974-2.
Deferred pediatric study under PREA for the treatment of poisoning by organophosphorous nerve agents as well as organophosphorous insecticides in pediatric patients ages birth to less than 17 years.
The final study report milestone was missed. Deferral Extension Requested December 22, 2014. Denied per FDA letter dated February 6, 2015. Deferral Extension Request resubmitted March 26, 2015. Denied per FDA letter dated May 7, 2015.Proposed p
DuoDote® (Atropine and Pralidoxime Chloride) Auto-Injector
A commitment to evaluate mangafodipir's effects on blood coagulation in people.
GE noted in their submission dated May 12, 2005 that the product has been discontinued and depleted from the US market. PMR/PMC Release Letter drafted and under review.
To conduct a study in both adult and pediatric patients to verify and describe the clinical benefit of Cyanokit and to assess its safety when used as indicated.
Pediatric study of an amoxicillin extended-release formulation for the treatment of tonsillitis and/or pharyngitis secondary to Streptococcus pyogenes in pediatric patients two to less than 12 years of age.
Deferred Pediatric Studies Under PREA You have agreed to conduct studies to assess the safety and effectiveness of desvenlafaxine succinate as a treatment for Major Depressive Disorder in pediatric patients ages 7 to 17 (children and adolescent
Revised Schedule: In a letter dated 15 March 2013, the FDA granted a deferral extension to the deadline for submission of the final clinical study reports for PMC #1229-1. Reasons for revisions: Slow enrollment, delay in start of Phase 3 due to
Deferred pediatric study under PREA for the management of secondary hyperparathyroidism in pediatric patients ages 5 to 18 years with Stage 3 or 4 chronic kidney disease (CKD) not yet on dialysis. Final Report Submission: April 2006
The Final Report Submission milestone was missed because of ongoing discussion with FDA regarding study design. Deferral Extension Requested May 14, 2013. Denied per FDA letter dated June 27, 2013. A draft protocol was submitted for FDA review
Regarding the hepatic impairment study, you have agreed to evaluate the PK of balsalazide in hepatically-impaired patients if clinically-relevant results are obtained in the renal impairment study.
Determine what dose adjustments are appropriate for pediatric patients with smaller body surface areas.(Fax 11/8/96)
Submit the results of the final analysis of overall survival data from the randomized clinical trial of vandetanib 300 mg vs. placebo in medullary thyroid cancer (Study 58).
Conduct a PK/PD study in adolescents ages = 13 years to 17 years with moderate to severe symptoms of primary Restless Legs Syndrome.
The study has not yet begun but does not meet the criteria for delayed.
Conduct a double-blind, randomized, placebo-controlled, parallel group efficacy and safety evaluation trial in adolescents = 13 years to 17 years with moderate to severe symptoms of primary Restless Legs Syndrome.
The Protocol Submission Date milestone for this PREA PMR has been missed.
Conduct a fixed-dose, placebo-controlled, double-blinded study that examines multiple doses in late Parkinson's disease. The trial should identify a range of doses inclusive of the lowest effective dose and the lowest maximally effective therap
Longitudinal studies involving follow up of Patient Treatment Data Forms and placement of data into a registry for periodic analyses related to post- marketing drug safety and uses. a. Protocol submission: Within 6 months of the date of final a
Per AP Letter Aug 11, 2004, the protocol due was date Feb 11, 2005, 6 month after application approval. A 3 month extension was requested, submission dated July 31, 2006 Draft longitudinal study protocol submitted Nov. 10, 2006, PK review compl
Deferred pediatric study under PREA for the use of Palladone (hydromorphone hydrochloride extended-release) Capsules 12-, 16-, 24-, and 32-mg for the management of persistent, moderate to severe pain in opiate-tolerant pediatric patients (0-16
Per FDA letter dated 01/24/2017, this PMR has been released.
A commitment to perform a Phase 4 adrenal suppression study in pediatric patients with large areas of involved skin using ACTH stimulation.  This will be followed by an adrenal suppression study in adult patients with large areas of involved sk
Conduct and submit the results of a multicenter, randomized clinical trial confirming the clinical benefit of olaratumab in combination with doxorubicin in patients with soft tissue sarcoma that is not amenable to surgery or radiation.
Deferred pediatric studies under PREA in positron emission tomography (PET) imaging for assessment of abnormal glucose metabolism to assist in the evaluation of malignancy in patients with known or suspected abnormalities found by other testing
NDA request to WD received Feb 6, 2014, FDA WD Acknowledgement letter signed Feb 12, 2014, PMR Release pending publication of WD in the FR
Deferred pediatric study under PREA for the prevention of postoperative nausea and vomiting (PONV) in pediatric surgical patients ages 0 to <17 years. A PK and safety study to characterize the pharmacokinetics of Zuplenz (ondansetron) oral solu
The final protocol and study completion milestones were missed.
Deferred pediatric study under PREA for the prevention of PONV in pediatric surgical patients ages 0 to <17 years. An adequately powered, well-controlled, and randomized dose-response study to evaluate the safety and efficacy of Zuplenz (ondans
The study has not begun but does not meet the criterion for delayed.
Submit the final analysis of safety in the ongoing trial (Protocol NO25026:BRIM3) to provide the potential for new safety signals from longer duration of exposure.
Submit an analysis of secondary malignancies for the proposed adjuvant melanoma trial [G027826: Phase III, Randomized, Double-Blind, Placebo- Controlled Study of Vemurafenib (RO5185426) Adjuvant Therapy in Patients with Surgically-Resected, Cut
Follow-up for secondary malignancies from the planned papillary thyroid cancer trial [N025530: An Open-Label, Multi-Center Phase II Study of the BRAF Inhibitor RO5185426 in Patients with Metastatic or Unresectable Papillary Thyroid Cancer (PTC)
Conduct a drug interaction trial to evaluate the effect of a strong CYP3A inducer (e.g., rifampin) on the pharmacokinetics of vemurafenib.
A randomized, double-blind, placebo-controlled, parallel group, multicenter clinical trial of the effect of avanafil on spermatogenesis in healthy adult males and adult males with mild erectile dysfunction.
To conduct a field study to evaluate the efficacy and safety of levofloxacin in the event of an attack with the intentional release of Yersinia pestis in the United States.
Deferred requirement for development of an age appropriate formulation for Pertzye (pancrelipase) Delayed-Release Capsules: Develop an age appropriate formulation to allow for dosing to the youngest, lowest weight pediatric patients, including
Fulfilled per the FDA letter dated 10/6/16
A 10 year, observational study to prospectively evaluate the incidence of fibrosing colonopathy in patients with cystic fibrosis treated with Pertzye (pancrelipase) Delayed-Release Capsules in the U.S. and to assess potential risk factors for t
The final protocol milestone was 5/31/2013. Sponsor has to reach legal agreement to join the joint sponsor protocol.
An observational study to estimate the prevalence of antibody seropositivity to selected porcine viruses in patients taking Pertzye (pancrelipase) Delayed-Release Capsules compared with an appropriate control group.
A clinical trial to assess the risk of QT prolongation with sublingual buprenorphine, i.e., a thorough QT (tQT) trial. A comparison to methadone at typical treatment doses should be included. It is likely this trial will need to be conducted in
Protocol was submitted on September 30, 2011. It was discussed with the FDA on December 15, 2014. On May 12, 2015, Indivior submitted a revised protocol for FDA’s review and received a response from FDA on July 28, 2015. Indivior is currently
A multi-phase clinical trial in a hyperphosphatemic pediatric dialysis population, with a placebo-controlled dose-response phase, followed by an open-label titration and maintenance phase, followed by a placebo-controlled randomized withdrawal
Study status: FMCNA requested that the Agency release the study commitment on February 22, 2013. The study is terminated.
Deferred pediatric study under PREA evaluating the use of Lymphoseek guided lymphatic mapping and sentinel lymph node biopsy in the treatment of solid tumors in pediatric patients ages 1 month to 16 years of age.
Draft Protocol and Final Protocol submitted by PMR milestone due dates
Lymphoseek™ (Technetium Tc 99m Tilmanocept )
Develop and validate an appropriate analytical method for determining the individual component phytosterol content in Liposyn II 20% IV Fat Emulsion.
The final report due date has passed with no final report received.
Liposyn® II (Safflower Oil and Soybean Oil [Fat Emulsions])
An observational study to evaluate the validity and reproducibility of the Prescription Opioid Misuse and Abuse Questionnaire (POMAQ), which will be used to identify opioid abuse and misuse behaviors among participants who have chronic pain whi
Embeda™ (Morphine Sulfate and Naltrexone Hydrochloride)
Targiniq™ ER (Oxycodone Hydrochloride and Naloxone Hydrochloride)
An observational study to validate measures of prescription opioid Substance Use Disorder and addiction in patients who have received or are receiving opioid analgesics for chronic pain.
MALLINCKRODT INC THE PHARMACEUTICALS BUSINESS OF COVIDIEN
Three-month repeat dose toxicity and toxicokinetic study in neonatal rats with a 28-day recovery period to provide safety assessment of Syndros (dronabinol oral solution) for pediatric clinical studies.
Deferred study under PREA to evaluate the pharmacokinetics of Syndros (dronabinol oral solution) for the treatment of chemotherapy induced nausea and vomiting (CINV) in pediatric cancer patients who failed to respond adequately to conventional
Deferred pediatric study under PREA to evaluate the tolerability and efficacy of Syndros (dronabinol oral solution) for the treatment of chemotherapy induced nausea and vomiting (CINV) in pediatric patients who failed to respond adequately to c
Pre-/postnatal developmental toxicology study in rats exposed to Syndros (dronabinol oral solution) to assess the risk of neurotoxicity.
Assess apixaban pharmacokinetics and pharmacodynamics in approximately 50 pediatric subjects aged 0 to less than 18 years, who are at risk for a venous or arterial thrombotic disorder, to determine dosing requirements for subsequent studies in
Conduct a randomized, open-label, active-controlled, safety and descriptive efficacy study to assess apixaban treatment in 150 pediatric patients evaluable for efficacy and safety, aged 0 to less than 18 years, requiring anticoagulation for the
A registry to provide long term evaluation of the durability of the treatment effect of the procedure in at least 100 corneal crosslinking-treated subjects at 3 years with a pre-treatment diagnosis of post-refractive corneal ectasia.
Photrexa® Viscous (Riboflavin 5'-Phosphate), Photrexa® (Riboflavin 5'-Phosphate), and KXL™ System
Evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response as the primary endpoint) of ombitasvir, paritaprevir, ritonavir, dasabuvir (VIEKIRA XR™) in pediatric patients greater than 3 years of age with c
Viekira® XR (Dasabuvir, Ombitasvir, Paritaprevir, and Ritonavir)
Conduct a repeat dose, pharmacokinetic/pharmacodynamics (PK/PD) study evaluating Adlyxin (lixisenatide) in patients with type 2 diabetes ages 10 to 17 years (inclusive) that are insufficiently controlled with metformin and/or basal insulin. Sub
Conduct a randomized multicenter, active-controlled trial to evaluate the safety and tolerability of AVYCAZ (ceftazidime-avibactam) in children from 3 months to less than 18 years of age with cUTI. The dose for this study will be determined upo
Conduct a randomized, multicenter, active-controlled trial to evaluate the safety and tolerability of AVYCAZ (ceftazidime-avibactam) in children from 3 months to less than 18 years of age with cIAI. The dose for this study will be determined up
Conduct a trial to evaluate the pharmacokinetics, safety and tolerability of AVYCAZ (ceftazidime-avibactam) in children from birth to less than 3 months of age with late-onset sepsis.
Conduct a prospective study over a five-year period after the introduction of AVYCAZ (ceftazidime-avibactam) to the market to determine if decreased susceptibility to AVYCAZ (ceftazidime-avibactam) is occurring in the target population of bacte
Conduct a trial or submit data from the Phase 3 trial in cIAI to evaluate the pharmacokinetics, safety, and clinical outcomes in adult patients with baseline renal impairment (creatinine clearance of 50 mL/min or less) receiving AVYCAZ (ceftazi
A pharmacokinetic/dose ranging study to determine the pediatric doses of Sustol (granisetron) extended-release injection, for subcutaneous use, and a controlled clinical trial with an active comparator to evaluate the efficacy, safety, and tole
A 1-month, GLP juvenile animal toxicology study of Sustol (granisetron) extended-release injection, for subcutaneous use, conducted in a single species. This study must be completed and submitted for review before initiating pediatric studies i
A multi-center clinical trial to evaluate the dosing and safety of ferric citrate for the treatment of hyperphosphatemia in children ages six months to < 18 years with chronic kidney disease
A protocol is being drafted and will be submitted for Agency review 1Q2017 following completion of the definitive juvenile toxicology study.
Develop and validate sensitive and precise assays for the detection of anti-linaclotide antibodies, including IgM, IgG, and IgA, that may be present in the serum at the time of patient sampling. A summary of the validation exercise including su
The final report milestone was missed because of additional time needed to develop an appropriate assay. FDA determined that the applicant demonstrated good cause for the delay.
Provide safety and efficacy data from a 2-year (minimum) clinical study of at least two additional doses of Macugen below 0.3 mg.
The Division agreed that the sponsor could terminate the clinical study EPOI014 due to problems with evaluation of the lower doses. However, the Division did not agree to release PMC #3 until the results of the other commitments are submitted a
You have committed to conduct a pharmacokinetic and pharmacodynamic study to assess the drug interaction potential of apomorphine with alcohol and antihypertensives to include vasodilators (including short-and long-acting nitrates). Protocol Su
Conduct and submit a final study report for a large simple safety study to assess the major clinical outcomes of death, progression of liver disease, and cancer in a broad population of HBV-infected patients using entecavir compared to standard
To cooperate with U.S.-based public health agencies in evaluating data on the use of Levaquin (levofloxacin) in a large U.S. population for inhalational anthrax (post-exposure) prophylaxis, should an exposure occur. This includes long-term safe
Submit comparative LVEF and cardiac safety data for patients enrolled on the adjuvant renal cell carcinoma trial, E2805 titled "A Randomized, Double-Blind Phase III Trial of Adjuvant Sunitinib versus Sorafenib versus Placebo in Patients with Re
The trial has completed enrollment. The final report is expected in April 2017.
Deferred pediatric study under PREA for the treatment of active ulcerative proctitis in pediatric patients ages 12 to 17 years.
Per FDA letter dated 09/02/2016, this PMR has been released.
A prospective, observational pregnancy outcome study to include fetal and neonatal outcomes and maternal pregnancy complications following a pregnancy exposed to ulipristal acetate (e.g., in case of inadvertent administration to a woman with an
The final report milestone was missed, due to poor subject enrollment and the FDA determined that the applicant demonstrated “good cause” for the delay. The final study report was received on 10/17/2016 which was on time and according to th
A case-control study of pregnancy loss complications. This study will be conducted as an expansion of the pregnancy outcome study (described under 1673-1), if a signal of concern regarding pregnancy complications is found in that study.
The final report milestone was missed, because PMR 1673-2 is contingent upon finding a safety signal in PMR 1673-1 and the FDA determined that the applicant demonstrated “good cause” for the delay.
An evaluation of the potential carcinogenicity of DMSO in a 2-year bioassay in the rat.
An evaluation of Fertility and Early Embryonic Development in a single species with DMSO.
An evaluation of Peri-and Postnatal Development in a single species with DMSO.
A clinical pharmacology study to assess pharmacokinetic parameters related to a Belviq dose of 10 mg in pediatric patients ages 7 to 11 years (inclusive). Data from this study should be considered when choosing dose(s) for the safety and effica
Per FDA letter dated 06/20/2016, this PMR has been fulfilled.
A 52-week, randomized, double-blind, placebo-controlled pediatric study to evaluate the safety and efficacy of Belviq for the treatment of obesity in pediatric patients ages 12 to 17 years (inclusive). You may not initiate this study until the
The protocol finalization milestone was missed because of ongoing discussion with FDA regarding study design.
A 52-week, randomized, double-blind, placebo-controlled pediatric study to evaluate the safety and efficacy of Belviq for the treatment of obesity in pediatric patients ages 7 to 11 years (inclusive). You may not initiate this study until resul
Provide clinical information from a 1-year (minimum) clinical study to support that there are no adverse effects on the corneal endothelium following the intravitreal administration of Macugen.
This study titled “Provide clinical information from a (minimum) 1-year study to support that there are no adverse effects on the corneal endothelium following intravitreal administration of Macugen.” As of December 2015, the study has over
Regarding the requested renal impairment study, you have agreed to compare the PK data from the multiple-dose PK study to that obtained from literature on patients with varying degrees of renal impairment that are receiving mesalamine or relate
BioMarin commits to designing and implementing a long-term study designed to assess growth and neurocognitive development with treatment with Kuvan (sapropterin dihydrochloride) in patients who are eight years of age or younger at study entry.
Zeneca is in the process of gathering and assessing clinical information about the use of propofol in ICU Sedation in children. During the past year, Zeneca has conducted two retrospective post-marketing surveys regarding the use of propofol in
With respect to requested in vivo drug interaction studies, based on the results of the in vitro drug interaction studies, firm agreed to conduct in vivo drug interaction studies in appropriate animal species and evaluate the PK (as deemed nece
Deferred clinical efficacy and safety study: A Pivotal, Randomized, Double-Blind, Controlled, Efficacy and Safety Study of the Use of Cycloset for the Treatment of Type 2 Diabetes Mellitus in Children Aged 10 - 16 years with a Diagnosis of Type
The final protocol due date has passed with no final protocol received.
The Study Completion milestone was missed because of difficulties in subject recruitment and enrollment; FDA determined that the applicant demonstrated sufficient justification for the delay. Original Final Report Due Date: 09/30/2011; Deferral
Janumet® (Metformin Hydrochloride and Sitagliptin Phosphate}
Sponsor commits to determine if body size adjustments are more appropiate for dosing in the pediatric population.
Commitment to conduct a study to investigate the use of this agent in the pediatric population.
PMC not fulfilled letter signed on 8/25/2010.
A dose finding, pharmacokinetics and safety trial in subjects with moderate to severe plaque psoriasis between the ages of 6 to 17 years.
As of the cutoff date, 3/20/16, 3 of the planned 32 subjects have been enrolled
A safety and efficacy trial in pediatric subjects with moderate to severe plaque psoriasis between the ages of 6 to 17 years.
Conduct a fixed-dose, placebo-controlled, double-blinded study that examines multiple doses in early Parkinson's disease. The trial should identify a range of doses inclusive of the lowest effective dose and the lowest maximally effective thera
You are required to assess the safety and effectiveness of Geodon as a treatment for bipolar disorder in pediatric patients  ages 10 to 17 (children and adolescents). Final Report Submission: February 11, 2008
The study has been initiated.85 patients out of the planned 180 patients have been enrolled into the trial.
Deferred pediatric study under PREA for the treatment of relief of mild to moderate acute pain in pediatric patients ages 1 to 2 years.
Deferral Extension Requested 07/28/2016.  Denied per FDA letter dated 09/16/2016.
Deferred pediatric study under PREA to improve wakefulness in patients with excessive sleepiness associated with narcolepsy in pediatric patients ages 6 to 17.
A total of 34 patients were enrolled in the study, no patients are ongoing in the study, 4 patients withdrew from the study, and 30 patients completed the study.
A 10-year, observational study to prospectively evaluate the risk of transmission of selected porcine viruses in patients taking Zenpep (pancrelipase) Delayed-Release Capsules.
A randomized trial of maintenance treatment with pralatrexate in previously untreated patients with PTCL who have demonstrated a response to CHOP or a CHOP-like regimen. Description of trial: This will be a Phase 3 multi-center, randomized clin
Deferred pediatric study under PREA for the prevention of nausea and vomiting in pediatric cancer patients ages 4 to <17 years receiving highly emetogenic chemotherapy (HEC). A PK and safety study to characterize the pharmacokinetics of Zuplenz
A 24-month oral (gavage) carcinogenicity study of glycopyrrolate in mice.
A 24-month oral (gavage) carcinogenicity study of glycopyrrolate in rats.
An assessment and analysis of spontaneous reports of injection site reactions associated with Vivitrol. Following approval, and according to the following timetable, submit the reports (containing both interval-based and comprehensive data) ana
An efficacy and safety, dose-finding study in children 12 months and older, weighing less than 25 kg, with secondary hypertension.
Conduct a controlled clinical trial to evaluate the efficacy of roflumilast as an add-on therapy to a long-acting beta agonist and inhaled corticosteroid fixed-dose combination therapy in the population of COPD patients for which roflumilast is
2354 patients enrolled of the total 2300 planned for enrollment
Deferred pediatric study under PREA: a 24-month, randomized, active-controlled, parallel group study to evaluate the single and multiple dose pharmacokinetics of fingolimod, and the safety and efficacy of multiple doses of fingolimod compared t
The final report submission milestone was missed because of delays involving organization of a large multicenter international trial and additional time required to enroll a larger number of trial subjects than originally planned. Original Fina
A postmarketing observational prospective, parallel cohort study in relapsing multiple sclerosis patients to assess the potentially serious risk of: eye toxicity, cardiac and vascular toxicity, pulmonary toxicity, seizures, serious and opportun
Develop and maintain a prospective, observational pregnancy exposure registry study conducted in the United States that compares the maternal, fetal, and infant outcomes of women exposed to fingolimod during pregnancy to an unexposed control po
A prospective, randomized, controlled study of fingolimod 0.5 mg, fingolimod 0.25 mg, and an appropriate control, of at least one year duration, to evaluate the efficacy and safety of the drug.
The final report milestone was missed because of difficulties in recruitment.
In order to support safe use of clonidine in combination with stimulants in pediatric patients and to provide additional safety information for labeling, you must conduct a juvenile animal study of clonidine in combination with a stimulant as a
Per FDA letter dated 08/16/2016, this PMR has been fulfilled.
Conduct a prospective 5-year pre-OLT (orthotopic liver transplant) registry study to collect and analyze data regarding renal function in patients with chronic hepatitis B and decompensated liver disease treated with VIREAD® (tenofovir disopro
A randomized trial comparing lapatinib in combination with trastuzumab and an aromatase inhibitor versus trastuzumab in combination with an aromatase inhibitor versus lapatinib in combination with an aromatase inhibitor in postmenopausal women
Difficulties in recruiting patients despite initiatives to increase enrollment.
Conduct a prospective, 3-arm trial evaluating HalfLytely with 5 mg bisacodyl, 2L polyethylene glycol solution plus electrolytes without bisacodyl, and 4L polyethylene glycol solution plus electrolytes without bisacodyl. The trial should evaluat
An adequate, placebo-controlled, double-blind, randomized, add-on design, superiority study to evaluate the safety and efficacy of a single dose of intravenous fosaprepitant, in combination with a 5HT3 antagonist, as compared to standard therap
Per FDA letter dated 10/13/2016, this PMR has been released.
A randomized, double-blind, adequately controlled study of efficacy, pharmacokinetics and pharmcodynamics of IV APAP for the treatment of acute pain in pediatric patients from 0 to 2 years of age.
A prospective, observational study in the United States, which includes a five year period of time after introduction of the TOBI Podhaler to the market to determine if decreased susceptibility to tobramycin is increasing in Pseudomonas aerugin
A pharmacokinetic analysis to determine a dosing regimen in children from 4 years to less than 16 years of age that provides drug exposure that is similar to the exposure that is effective in adult patients with partial onset seizures. This ana
A pharmacokinetic and safety analysis in children from 1 month to less than 16 years of age to determine whether the bioavailability of the intravenous and oral formulations is similar and to determine an acceptable safety margin of the intrave
A one year, prospective observational cohort study in the United States of CF patients chronically colonized with P. aeruginosa who use TOBI Podhaler as part of their regular care compared to patients using other FDA approved inhaled antipseudo
An actual use human factors study to validate the approved Instructions for Use (IFU). The study will enroll 45 patients in total with three age groups of 15 patients each: 6-10 years, 11-17 years, and > 18 years. Only CF patients naïve to use
Create adjunct instructions for use using alternative media and validate these instructions for use to ensure the patient can safely and effectively perform the critical tasks for the intended use of this product.
Final protocol submission date of May, 2014 was not met. The Sponsor submitted the protocol on 12/21/2015, according to the new timeline they proposed in 2014.
To modify acceptance criteria for the current peptide mapping procedure to include selected peak area and retention times.
Conduct a Phase 3 randomized controlled trial in 1222 patients with hormone-refractory metastatic prostate cancer previously treated with docetaxel comparing cabazitaxel 20 mg/m2 with prednisone versus cabazitaxel 25 mg/m2 with prednisone and p
In order to verify the clinical benefit of eteplirsen, conduct a 2-year randomized, double-blind, controlled trial of eteplirsen in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Patients should be
Deferred pediatric study under PREA for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 16 years (inclusive).
Conduct a prospective, randomized clinical trial to demonstrate safety and effectiveness of Dificid (fidaxomicin) compared to vancomycin in pediatric patients (6 months to less than 18 years of age) with C. difficile-associated diarrhea.
Conduct a prospective study over a five-year period after introduction of Dificid (fidaxomicin) to the market to determine if decreased  susceptibility to Dificid (fidaxomicin) is occurring in C. difficile. Provide a detailed protocol describin
Conduct a prospective, randomized, comparative trial to demonstrate the efficacy of Dificid (fidaxomicin) in the treatment of patients with multiple recurrences of C. difficileassociated diarrhea.
Applicant to conduct a Phase 4 study to confirm the clinical benefit of midodrine. Protocol drafts submitted at 7/18/96 meeting.  Protocols are 401, 402 and 403.
Deferred study under PREA to evaluate the pharmacokinetics, healing, maintenance of healing, and symptoms of endoscopy-proven erosive esophagitis (EE) in patients 12 years to 17 years of age.
Deferred pediatric study under PREA for the maintenance of remission of ulcerative colitis in pediatric patients 5 to 17 years of age.
Conduct a randomized, dose-exploration, multicenter clinical trial evaluating the multiple dose PK/PD profile and safety of oral rivaroxaban (tablets or oral suspension) in pediatric patients aged 6 months to < 6 years with VTE.
35 subjects have been treated subjects: 7 (6 months to < 2 years), 28 (2 to < 6 years) since Jan 2015. Trial completion is due December 2019.
Conduct a single-dose PK/PD and tolerability trial in pediatric patients age birth to < 6 months with VTE to determine doses of rivaroxaban (oral suspension) that provide similar exposure and/or PD effect to those seen in older pediatric cohort
3 subjects have been treated since Nov 2015. Trial Completion is due 12/2019
Conduct a dose-exploration, multicenter clinical trial evaluating the multiple dose PK/PD profile and safety of oral rivaroxaban (oral suspension) in pediatric patients aged birth to <6 months with VTE.
A deferred pediatric study under PREA for the treatment of schizophrenia in pediatric patients ages 13 to 17 years. A study to obtain pharmacokinetic data and provide information pertinent to dosing of lurasidone tablets in the relevant pediatr
A deferred pediatric study under PREA for the treatment of schizophrenia in pediatric patients ages 13 to 17 years. A study of the efficacy and safety of lurasidone tablets in the relevant pediatric population.
It is not apparent from the trials you have conducted in schizophrenia that the lowest effective dose of lurasidone has been identified. We ask that you further characterize the utilization of lurasidone in the treatment of adults with schizoph
Conduct a randomized, single-blind, multicenter dose ranging study comparing the safety and efficacy of SUPREP to NuLytely in children (6 months to 2 years).
Suprep® Bowel Prep Kit (Sodium Sulfate, Potassium Sulfate, and Magnesium Sulfate)
A long term ( 24 week, 3on/off cycle) postmarketing observational study in 50 patients describing the safety and tolerability of Bethkis in patients with a stable FEV1 >/= 25 to <40% predicted. The following efficacy outcomes should also be col
The Sponsor did not meet the original timelines, new timelines acknowledged on June 23, 2015
Deferred randomized and controlled pediatric study under PREA to evaluate efficacy, safety, and pharmacokinetics of saxagliptin for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 16 years.
The study was initiated 2nd quarter of 2011. Original Final Report Due Date: 06/30/2015; Deferral Extension granted per FDA letter dated 05/30/2015. The deferral extension was granted because of difficulties experienced in recruiting study part
A randomized, double-blind, controlled trial evaluating the effect of saxagliptin on the incidence of major adverse cardiovascular events in patients with type 2 diabetes mellitus.
A carcinogenicity study of orally administered clobazam in rats.
A carcinogenicity study of orally administered clobazam in mice.
Deferred pediatric study under PREA for the treatment of endoscopically diagnosed esophagitis, including erosive and ulcerative esophagitis and associated heartburn due to GERD for up to 8 weeks in pediatric patients ages birth to 12 years.
The final study report milestone was missed. The sponsor has submitted withdraw of this NDA and will not be conducting this study.
Submit the per protocol overall survival follow-up (data cut-off date will be at least 5 years from the date of the last patient randomized) for Trial SSGXVIII/AIO entitled "Short (12 months) versus long (36 months) duration of adjuvant treatme
A thorough QTc clinical trial in patients treated with Tygacil (tigecycline).
Deferred pediatric trial under PREA: A randomized, controlled, parallel group superiority trial to evaluate the single and multiple dose pharmacokinetics of teriflunomide, and the safety and efficacy of teriflunomide compared to an appropriate
As of September 15, 2015, 38 of the 165 patients have been enrolled into the trial.
A prospective, observational exposure cohort study conducted in the United States that compares the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to teriflunomide during pregnancy to unexposed control populations
A pharmacokinetic study in pediatric patients with partial-onset seizures aged 1 month to < 24 months. At least 2 maintenance dose levels of FYCOMPA (perampanel) should be evaluated to characterize pharmacokinetic parameters following multiple
The final protocol milestone was missed due to re-evaluation of pediatric dosing and harmonization of milestones with concurrent PMR study.
A pharmacokinetic study in pediatric patients with partial-onset seizures aged 2 to < 12 years. At least 2 maintenance dose levels of perampanel should be evaluated to characterize pharmacokinetic parameters following multiple administration of
The final report milestone was missed. Original Final Report Due Date: 5/31/14. Per FDA letter dated 7/1/15, final report due date extended to 7/31/16.
It is not apparent from the studies you have conducted in bipolar mania that the lowest effective dose of asenapine has been identified. We request that you further characterize the utilization of asenapine in the treatment of adults with acute
A randomized, double-blind, controlled trial evaluating the effect of Victoza (liraglutide [rDNA origin]) injection on the incidence of major adverse cardiovascular events in patients with type 2 diabetes mellitus. This trial must also assess a
The Final Report Submission milestone was missed because trial initiation was delayed due to protocol discussion with FDA and additional analyses were required that were expected to lengthen the report preparation time between trial completion
Deferred pediatric study under PREA for the prevention of nausea and vomiting in pediatric cancer patients ages 4 to <17 years receiving HEC. An adequately powered, well-controlled, and randomized dose-response study to evaluate the safety and
Test for the individual component phytosterol content in all batches of Liposyn II 20% IV Fat Emulsion, manufactured over a three year period, using the method developed under PMR 2142-1. Based on these test results, establish limits for each o
Perform phenotypic resistance testing of neuraminidase activity and direct genotypic resistance testing of neuraminidase and hemagglutinin in a study to assess the safety and tolerability of oseltamivir when administered as treatment for influe
The trial has not been completed because additional patient recruitment is required to complete the trial. FDA issued an acknowledge revised milestones and sufficient justification for anticipated delay of postmarketing requirement milestones l
A clinical trial to assess the risk of QT prolongation with Subutex sublingual tablets, i.e., a thorough QT (tQT) trial. A comparison to methadone at typical treatment doses should be included. It is likely this trial will need to be conducted
A clinical trial to assess the risk of QT prolongation with Suboxone sublingual tablets, i.e., a thorough QT (tQT) trial. A comparison to methadone at typical treatment doses should be included. It is likely this trial will need to be conducted
Conduct a 3 - arm randomized trial investigating the combination of everolimus with exemestane versus everolimus alone versus capecitabine in patients with estrogen - receptor positive metastatic breast cancer after recurrence or progression on
Conduct a randomized, controlled trial to evaluate the risk of major adverse cardiac events with aclidinium bromide in patients with COPD.
A clinical pharmacology trial to assess pharmacokinetic and pharmacodynamic parameters related to Qsymia doses of 3.75 mg/23 mg, 7.5 mg/46 mg, 11.25 mg/69 mg, and 15 mg/92 mg in pediatric patients ages 12 to 17 years (inclusive). Data from this
The trial completion milestone was missed because the required juvenile animal study data were under review by the Agency until November 6, 2015. FDA determined that the applicant demonstrated sufficient justification for the delay. Original Fi
A 52-week randomized, double-blind, placebo-controlled pediatric trial to evaluate the safety and efficacy of Qsymia for the treatment of obesity in pediatric patients ages 12 to 17 years (inclusive). This trial should not be initiated until af
The Final Protocol Submission milestone was missed, because of ongoing discussion with FDA regarding the study design and FDA determined that the applicant demonstrated sufficient justification for the delay. Original Final Report due date: Sep
A clinical pharmacology trial to assess pharmacokinetic and pharmacodynamic parameters related to Qsymia doses of 3.75 mg/23 mg, 7.5 mg/46 mg, 11.25 mg/69 mg, and 15 mg/92 mg in pediatric patients ages 7 to 11 years (inclusive). Data from this
A 52-week randomized, double-blind, placebo-controlled pediatric trial to evaluate the safety and efficacy of Qsymia for the treatment of obesity in pediatric patients ages 7 to 11 years (inclusive). You may not initiate this trial until result
Forty-five subjects have been enrolled in a pediatric pharmacokinetic study and 74 subjects have been enrolled in an open-label safety and tolerability extension study. Twenty-three subjects are enrolled in a long-term pediatric efficacy and sa
Perform a trial assessing the CSF concentration profile of Teflaro (ceftaroline fosamil) in infants < 2 months of age. A minimum of 12 infants < 2 months of age receiving antibacterials for treatment of late-onset neonatal sepsis must be studie
Perform a randomized comparison of Teflaro (ceftaroline fosamil) and comparator in infants < 2 months of age with ABSSSI and CABP including patients with infections suspected or demonstrated to be caused by MRSA.
Conduct a prospective study over a five-year period after introduction of Teflaro (ceftaroline fosamil) to the market to determine if decreased susceptibility to Teflaro (ceftaroline fosamil) is occurring in the target bacteria included in the
A bioequivalence trial of Prestalia (perindopril and amlodipine) and ACEON (perindopril erbumine) in healthy volunteers. The proposed trial will provide an accurate estimate of the relative bioavailability of perindopril and the active metaboli
Prestalia® (Perindopril Arginine and Amlodipine Besylate)
Submit the final report for the pediatric pharmacokinetic trial entitled “Pharmacokinetics of SFP iron delivered via dialysate in pediatric patients with chronic kidney disease on hemodialysis.”
The study was initiated on September 30, 2015. Three sites of seven have been initiated and enrolled patients. The trial Completion is due February 28, 2017
Efficacy and safety trial of Triferic via hemodialysate in pediatric patients aged less than 18 years with hemodialysis-dependent chronic kidney disease.
Final Protocol Submission is due March 31, 2018
Conduct a study to evaluate the safety and efficacy of secukinumab in pediatric subjects ¡Ý 6 years of age with plaque psoriasis.
The trial is ongoing. Trial Completion is not due until March 2016
Deferred pediatric study of pharmacokinetics and safety under PREA for the management of mild to moderately severe pain when the use of an opioid analgesic is appropriate in pediatric patients ages 2 to 17 years.
Peds study final study report due in 2018
Deferred pediatric study of pharmacokinetics, safety and efficacy under PREA for the management of mild to moderately severe pain when the use of an opioid analgesic is appropriate in pediatric patients ages one month to 2 years.
A postmarketing pharmacovigilance study of the risk factors, clinical management, and outcome of cases of major bleeding in association with Xarelto (rivaroxaban) use.
A study to determine the feasibility of conducting nonclinical (embryo-fetal development, prenatal and postnatal development, carcinogenicity) studies to assess the potential toxicity of 9 desmethyl-beta dihydrotetrabenazine (1-O-dealkyl DHTBZ,
A randomized controlled trial in patients with histologically documented, advanced or recurrent (Stage IIIB and not amenable for combined modality treatment) or metastatic (Stage IV) non-small cell lung cancer (NSCLC) who have not experienced d
Conduct a randomized, single-blind, multicenter dose-ranging study comparing the safety and efficacy of Prepopik to community standard of care in children (ages 9 years to 16 years). This study will include PK assessments.
The study is recruiting patients. Original Final Report Due Date: 8/31/16; Deferral Extension granted per FDA letter dated 5/27/16.
Prepopik™(Sodium Picosulfate, Magnesium Oxide, and Citric Acid)
Conduct a randomized, single-blind, multicenter dose-ranging study comparing the safety and efficacy of Prepopik to community standard of care in children (ages 2 years to <9 years). This study will include PK assessments.
Conduct a randomized, single-blind, multicenter dose-ranging study comparing the safety and efficacy of Prepopik to community standard of care in children (ages 12 months to <2 years). This study will include PK assessments.
Conduct a retrospective study to identify the risk factors associated with development of persistent deterioration of renal function in patients undergoing colon cleansing with Prepopik in preparation for colonoscopy.
Conduct a randomized, active-controlled, multicenter clinical trial evaluating the safety, efficacy and PK/PD (sparse sampling) of at least 3 months of treatment with oral rivaroxaban (tablets or oral suspension) in pediatric patients aged birt
168 subjects have been treated: 113 (12 to < 18 years), 55 (6 to < 12 years) since Nov 2014. Trial Completion is due 12/2022.
Conduct a confirmatory randomized double blind placebo controlled multicenter Phase 3 trial in subjects with sputum smear-positive pulmonary multidrug resistant tuberculosis (MDR-TB). This trial should assess long term outcomes of failure or re
Develop a patient registry for bedaquiline-treated patients to assess incidence rates of serious adverse events, including death. The registry should capture the information listed below: a. indication for use, including utilization of expert m
Conduct a prospective in vitro study over a five-year period after introduction of SIRTURO (bedaquiline) to the market to determine MICs of MDR-TB isolates to bedaquiline for the first 5 years from marketing. Report interpretation of these MICs
A clinical pharmacology study to evaluate the pharmacokinetics, pharmacodynamics, and safety of canagliflozin in pediatric patients ages 10 to <18 years with type 2 diabetes mellitus on metformin monotherapy.
Invokamet™ (Canagliflozin and Metformin Hydrochloride)
A 26-week, randomized double-blind, placebo-controlled study, followed by a 26-week double-blind, placebo- or active-controlled extension, to evaluate the efficacy and safety of canagliflozin compared to placebo in pediatric patients ages 10 to
The trial has not begun but does not meet the criterion for delayed
An assessment and analysis of all foreign and domestic spontaneous reports of malignancy (pheochromocytoma, Leydig cell tumor, and renal cell carcinoma), fatal pancreatitis, hemorrhagic/necrotizing pancreatitis, severe hypersensitivity reaction
Deferred pediatric study under PREA for the treatment of sedation of non-intubated patients prior to and/or during surgical and other procedures in pediatric patients 0 to 16 years of age.
Original Final Report Due Date: 12/31/2012. Per FDA letter dated 12/02/2013, final report due date extended to 08/31/2015. Second Deferral Extension Granted per FDA letter dated 12/29/2016.
A study analyzing data from the Registry provided for in the REMS to evaluate the development of visual lesions, timing and risk of the development of concentric field loss, the risk of visual acuity deficits, the potential for progression of t
A 10 year, observational study to prospectively evaluate the incidence of fibrosing colonopathy in patients with cystic fibrosis treated with PANCREAZE (pancrelipase) Delayed-Release Capsules in the U.S. and to assess potential risk factors for
A 10 year, observational study to prospectively evaluate the risk of transmission of selected porcine viruses in patients taking PANCREAZE (pancrelipase) Delayed-Release Capsules.
Conduct a clinical trial of patients with progressive, locally advanced or metastatic, treatment-naive or previously treated pancreatic neuroendocrine tumors (pNET) to verify and describe the efficacy and safety of sunitinib.
A prospective cohort study to a) determine the frequency of pregnancy in women of child-bearing age prescribed Qsymia and b) compare the risk of oral clefts, major congenital malformations, and low birth weight in offspring of women exposed to
A drug-use study conducted annually for 7 years with nationally representative and projected data to provide a denominator in order to assess adverse event reporting rates of the known serious risk of congenital malformation (specifically orofa
A randomized, placebo- and active-controlled trial to evaluate changes in renal function in obese adults, who will be randomized to Qsymia (3 dosage strengths) or placebo. The primary objective of the trial will be to assess the change in measu
A randomized, double-blind, placebo-controlled trial to evaluate the effect of long-term treatment with Qsymia on the incidence of major adverse cardiovascular events (non-fatal myocardial infarction, non-fatal stroke, and cardiovascular death)
The Final Protocol Submission milestone was missed, because of ongoing discussion with FDA regarding the study design and FDA determined that the applicant demonstrated good cause for the delay.
Conduct a clinical trial (PX-171-007) to evaluate the safety of a 30-minute intravenous infusion of carfilzomib at the dose of 20/56 mg/m squared in patients with multiple myeloma
ONYX THERAPEUTICS INC A WHOLLY OWNED SUB OF AMGEN INC
Conduct a clinical trial in patients with hepatic impairment to assess safety and PK characteristics of carfilzomib administered as a 30 minute infusion. The number of patients enrolled in the trial should be sufficient to detect PK differences
Conduct one or more clinical trials including Phase 3 Protocol 2011-003, supplemented as needed by an additional trial, to evaluate the PK, safety, and efficacy of carfilzomib in patients with varying degrees of renal impairment and those on ch
An assessment and analysis of spontaneous reports of serious hepatic abnormalities, fatal pancreatitis, hemorrhagic/necrotizing pancreatitis, and severe hypersensitivity reactions (angioedema, anaphylaxis, Stevens Johnson Syndrome) in patients
The first interim report submission due on March 31, 2014, was missed. The applicant proposed revised milestones to harmonize submission of these interim reports with PSURs reported annually in June. FDA determined that the applicant demonstrat
Kazano® (Alogliptin and Metformin Hydrochloride)
A single-dose, open-label, pharmacokinetic trial in breastfeeding women receiving fospropofol for a needed procedure. Concentrations of Lusedra will be assessed in breast milk so as to estimate potential infant exposure.
You will perform a "Thorough QT Study" as described in ICH E14. This study can utilize your extended release formulation or an immediate release formulation. It can utilize a single 0.6 mg dose group and a single administration.
The drug product application Clonidine ER Oral Suspension has been discontinued. Accordingly, at the present time the above stated commitments remain unmet. If the drug product application, Clonidine ER Oral Suspension, is reinstated and become
A carcinogenicity study in one animal species in which plasma levels of 9-desmethyl-beta-dihydrotetrabenazine can be achieved that are similar to or greater than plasma levels in humans at clinically relevant doses of tetrabenazine.
A one year prospective, randomized, parallel, active-control arm trial to compare the safety of Topamax (topiramate) with regard to metabolic acidosis, renal stone formation, bone mineral density, and development (i.e. height, weight and sexual
A safety and efficacy study in pediatric patients with irritable bowel syndrome with constipation ages seven years up to 17 years treated with Linzess (linaclotide).
9 patients out of the planned 260 patients have been enrolled into the trial.
A clinical trial in adults receiving Linzess (linaclotide) to assess development of antidrug antibody (ADA) responses in patient samples. Validated assays capable of sensitively detecting ADA responses that may be present at the time of patient
Submit the results of a single-dose pharmacokinetic (PK)/pharmacodynamic (PD) and tolerability trial in pediatric patients aged >/= 6 months to < 17 years with venous thromboembolic event(s) (VTE) in appropriate age cohorts to determine dosing
Final Study Report submitted March 11, 2016
Conduct a randomized, dose-exploration, multicenter clinical trial evaluating the multiple dose PK/PD profile and safety of oral rivaroxaban (tablets or oral suspension) in pediatric patients aged 6 years to <17 years with VTE. Enroll into sequ
64 subjects have been treated: 24 (12 to < 18 years), 40 (6 to < 12 years) since Feb 2013. Trial completion is due 12/2016.
Longer duration follow-up: Continue follow-up of patients (on treatment and in protocol defined post-treatment follow-up) and submit a final analysis report of trial AP24534-10-201 with 24 months of minimum follow-up for each patient. If 24 mon
A randomized, double-blind, placebo-controlled trial evaluating the effect of canagliflozin on the incidence of major adverse cardiovascular events (MACE) in patients with type 2 diabetes mellitus. The primary objective of the trial should be t
Deferred pediatric study under PREA to assess efficacy of tramadol in pediatric patients 7 to less than 17 years of age.
Deferred pediatric study under PREA to assess the pharmacokinetics, safety and tolerability of tramadol in pediatric patients 7 to less than 17 years of age.
Conduct a trial in 100 evaluable pediatric patients with plaque psoriasis ages 2 to 16 years and 11 months. Evaluate the safety and effect of Topicort (desoximetasone) Topical Spray, 0.25% on the hypothalamic-pituitary-adrenal axis and pharmaco
The final report milestone was missed because of slow enrollment. The applicant has not requested a deferral extension.
Deferred pediatric study under PREA to evaluate the safety of SITAVIG (acyclovir) in pediatric patients greater than 6 years to less than 18 years of age with recurrent herpes labialis and to assess duration of herpes simplex virus (HSV) episod
Per FDA letter dated 07/12/2016, this PMR has been released.
A 2-year carcinogenicity study in a single species.
A registry of patients, including infants, with NAGS deficiency being treated with carglumic acid to obtain long-term clinical safety information. Data to be collected will include patient demographics, details of treatment with carglumic acid,
Conduct a controlled trial (trial of pediatric HBV-infected subjects required under PREA) that elucidates the mechanism of tenofovir's effects on bone. Evaluations of adequate numbers of pediatric subjects must include the following: a. Measure
Conduct a Phase 1/2 single arm clinical trial to investigate the pharmacokinetic, safety, and preliminary efficacy of omacetaxine following fixed dose  administration in patients with chronic phase (CP) or accelerated phase (AP) chronic myeloid
The Sponsor is still having difficulty with study enrollment. The original final report was due 6/2016. The 2013 revised final report was due 12/2016
Conduct a mass balance trial in humans to determine the disposition and elimination pathways as well as to characterize the major metabolites of omacetaxine following subcutaneous injection. Depending on the results, hepatic and/or renal impair
Conduct an observational clinical study in pediatric patients treated with Kalydeco (ivacaftor) to evaluate the risk of cataracts. The study should include eye examinations by an ophthalmologist to assess for both visual acuity and cataracts/ey
A rodent carcinogenicity study in the rat designed according to "FDA Guidance for Industry-Carcinogenicity Study Protocol Submissions". Submit the carcinogenicity protocol for Special Protocol Assessment prior to initiating the study.
A study of the effects of carglumic acid on pregnancy and fetal outcomes. This study can be performed as a sub-study within the registry for all patients with NAGS deficiency. Information on pregnancy and fetal outcomes should be submitted annu
A long-term prospective observational study (product exposure registry) of patients with homozygous familial hypercholesterolemia (HoFH) treated with Kynamro (mipomersen sodium) to evaluate known and potential serious risks related to the use o
An assessment and analysis of spontaneous reports of serious hepatic abnormalities, malignancy, and immune-mediated reactions in patients treated with Kynamro (mipomersen sodium) for a period of 10 years from the date of approval. Specialized f
Conduct a drug interaction trial to evaluate the effect of a strong CYP3A4 inhibitor (e.g., ketoconazole) on the pharmacokinetics of vemurafenib.
Sponsor submitted new study protocol 17- MAR-15 and on 26-MAY-15, FDA requested revisions. Clinical trial commitment status is pending.
Conduct a clinical trial in patients with normal hepatic function and patients with pre-existing severe hepatic impairment to assess the effect of severe hepatic impairment on the pharmacokinetics of vemurafenib.
Develop an Investigational Use Only, Companion Diagnostic (IUO CoDx) that reliably detects V600K BRAF mutation in patients with unresectable or metastatic melanoma and conduct an open-label single arm trial with overall response rate and durati
Deferred pediatric study under PREA for the treatment or prevention of upper gastrointestinal bleeding in critically ill pediatric patients ages 2 to 11 years old and 12 to 16 years old.
Through collaboration with the Antiretroviral Pregnancy Registry, conduct a prospective observational study in order to collect and analyze data on maternal and fetal outcomes in 200 women who become pregnant while taking Truvada® for a pre-ex
Truvada® (Emtricitabine and Tenofovir Disoproxil Fumarate)
Collect and analyze data from individuals who take Truvada® for pre-exposure prophylaxis (PrEP) of sexually acquired HIV-1 infection and who seroconvert during follow-up. The following data should be collected and the following analyses conduc
The trial will be extended for two additional years to allow for potentially more data collection.
Conduct an analysis of data from ongoing and planned demonstration projects (trials) including at least 7000 uninfected individuals taking Truvada for a pre-exposure prophylaxis (PrEP) indication with the objective of examining the association
Milestone revised to have additional time for subject follow-up, data collection and reporting.
In the context of a U.S. Centers for Disease Control and Prevention (CDC) demonstration project (trial) for once-daily Truvada for a pre-exposure prophylaxis (PrEP) indication, validate an adherence questionnaire over the period of the demonstr
Milestone revised to have additional time to enroll adequate number of subjects
An 8-week randomized, double blind, placebo-controlled trial in children 5 to 17 years of age with active, mild to moderate ulcerative colitis. The trial will evaluate pharmacokinetics (PK), efficacy for induction of remission, and safety of at
Deferral Extension Requested 06/08/2016.  Denied per FDA letter dated 07/25/2016.
Deferred pediatric study under PREA for the management of fibromyalgia in pediatric patients ages 13 to 17.
Per FDA letter dated 12/22/2016, this PMR has been fulfilled.
Deferred pediatric study under PREA, a randomized, double-blind, placebo-controlled study to evaluate the efficacy, pharmacokinetics, and safety of pregabalin in pediatric patients with fibromyalgia ages 13 through 17 years, inclusive.
Examine patients 0-23 months of age who are referred for an MRI exam with contrast. A sufficient number of subjects will be studied to adequately characterize the pharmacokinetics of the product in this age group. At least 40 patients will be e
Final Study Report submitted June 3, 2016, prior to the milestone date. Guerbet's required PREA PMR pediatric assessment and labeling supplement for PMR 2021-2, which was deferred until June 30, 2016, hasn’t been submitted. Guerbet’s respon
A multiple-dose pharmacokinetic trial in children from 2 to less than 18 years of age with juvenile idiopathic arthritis (JIA)
The Final Report was submitted to FDA on 9/16/16
A randomized withdrawal, double-blind, placebo-controlled trial to evaluate the efficacy and safety of tofacitinib in children from 2 to less than 18 years of age with polyarticular-course juvenile idiopathic arthritis.
7 patients out of the planned 210 patients have been enrolled.
Controlled clinical trial to evaluate the long term safety of tofacitinib in patients with rheumatoid arthritis. The trial should include two doses of tofacitinib and an active comparator. The trial should be of sufficient size and duration to
3835 patients out of the planned 4000 have been enrolled. Final protocol milestone was missed because the draft protocol required significant modifications based upon the Agency’s recommendations and the FDA determined that the applicant demo
Conduct a study to assess the safety of Zutripro (hydrocodone, chlorpheniramine, and pseudoephedrine combination product oral solution) in approximately 400-450 children 6-17 years of age with symptoms of the common cold. The dose used in this
Original Final Report Due Date: 09/30/2016; Deferral Extension granted per FDA letter dated 07/20/2016
Zutripro® (Hydrocodone Bitartrate, Chlorpheniramine Maleate, and Pseudoephedrine Hydrochloride )
Conduct a study to assess the safety of Rezira (hydrocodone and pseudoephedrine combination product oral solution) in approximately 400-450 children 6-17 years of age with symptoms of the common cold. The study will be conducted with a formulat
Original Final Report Due Date: 09/30/2016; Deferral Extension granted per FDA letter dated 07/20/2016.
Rezira® (Hydrocodone Bitartrate and Pseudoephedrine Hydrochloride)
Conduct a prospective, randomized, placebo-control, double-blinded efficacy/safety trial of Potiga (ezogabine) in children >/=12 years old.
Original Final Report Due Date: 05/31/2018; Deferral Extension granted per FDA letter dated 11/25/2014.
GLAXOSMITHKLINE INTELLECTUAL PROPERTY MANAGEMENT LTD ENGLAND
Conduct a long-term open label extension study of ezogabine in children >/=12 years old.
The study was initiated on September 4, 2012 and was terminated on November 29. 2013 due to the imposition of a full clinical hold. At the time of the clinical hold four subjects had been enrolled.
An in vitro study to evaluate whether ezogabine is a substrate for major transporters in the kidney. Refer to the Agency's Guidance (http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM072101.pdf) for more de
A controlled urodynamic trial, to include adults of both sexes in a wide range of ages, including the elderly. Pre- and post-drug urodynamic measures should be carefully collected. Urodynamic measurements should include, although not necessaril
Study completion date of July 31, 2015 is delayed. Per sponsor, enrollment of investigators and patients was challenging before safety issues were identified and has become more challenging since implementation of additional monitoring. As of J
To perform a randomized, blinded, controlled trial of previously untreated PTCL patients randomized to treatment with CHOP or to romidepsin plus CHOP, with Progression Free Survival as the primary efficacy endpoint. Final Progression Free Survi
A large randomized, double-blind, active- and placebo-controlled trial to compare the risk of clinically significant neuropsychiatric events, including but not limited to suicidality, in individuals using Zyban (bupropion hydrochloride), vareni
Observational study to collect long-term safety data in at least 50 pediatric patients with chronic kidney disease on erythropoietin-stimulating agent (ESA) therapy who require iron maintenance treatment for iron deficiency anemia.
Assess the long-term efficacy (and safety) of Exjade® (deferasirox) treatment to a target LIC of 3 mg Fe/g dw followed by one or more treatment holidays until the LIC is >= 5 mg Fe/g dw in patients with NTDT. Follow-up of all subjects for up t
Conduct a trial to assess the long-term efficacy of Exjade® (deferasirox) in patients with NTDT and high LIC. The trial should assess response rates in the subset of patients with baseline LIC values >15 mg Fe/g dw (proportion of patients achi
Conduct a prospective, randomized trial in at least 210 patients with low to intermediate risk myelodysplastic syndromes (MDS) receiving Exjade® (deferasirox) for transfusional iron overload (approximately 140) or placebo (approximately 70) to
Establish a registry of children (aged 10 to <18 years old at enrollment) with NTDT and treated with Exjade® (deferasirox) for documented iron overload. Study 2422 will follow at least 40 children for up to 5 years to assess and analyze the lo
Conduct an enhanced pharmacovigilance study (including proactive surveillance and follow-up of spontaneous reports) to characterize the frequency and severity of adverse Events of Special Interest (ESIs), defined as all deaths and severe or ser
Assess the long-term safety of Exjade® (deferasirox) in patients with NTDT by conducting a trial of Exjade® (deferasirox) for the treatment of iron overload (LIC >=5 mg Fe/g dw) in non-transfusion dependent thalassemia (NTDT) in patients aged
Completion of the planned mass balance trial. Contingent on FDA review of the mass balance results, a clinical pharmacokinetic trial in patients with hepatic impairment may be required. Description of trial: This is an ongoing Phase 1 mass bala
Deferred pediatric study under PREA, a Phase 1 pharmacokinetic and safety study for the treatment of chronic pain in opioid tolerant pediatric patients ages 7 through 17 years.
Original Final Report Due Date: 11/30/2014; Deferral Extension granted per FDA letter dated 9/26/2014. Final Report due date extended to 04/30/2017. Original Study Start Date: 05/30/2012 extended to: 10/31/2014. Original Study Completion: 05/30
Deferred pediatric trial under PREA: A prospective, randomized, controlled, double-blind, efficacy/safety study of oxcarbazepine ER for the adjunctive treatment of partial onset seizures in children ages one month to < 2 years. The primary effi
Deferred pediatric trial under PREA: A prospective, randomized, controlled, double-blind, efficacy/safety study of oxcarbazepine ER for the adjunctive treatment of partial onset seizures in children ages 2 to <6 years. The primary efficacy endp
A clinical trial to examine pharmacokinetics and tolerability in children ages 6 months to 4 years using an age appropriate extended release oxcarbazepine formulation.
The final protocol submission milestone was missed because of difficulty in obtaining a suitable formulation.
A clinical trial to examine pharmacokinetics and tolerability in children, ages 1 month to 6 months, using an age appropriate extended release oxcarbazepine formulation.
To change the text on the backing film of the drug product to a darker ink within one year from the date of this approval letter
A 52-week, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of alogliptin when added on to metformin in pediatric patients ages 10 to 17 years (inclusive) with type 2 diabetes mellitus. At least 30% of rand
A 52-week, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of alogliptin in pediatric patients ages 10 through 17 years (inclusive) with type 2 diabetes mellitus . At least 30% of randomized subjects will
A randomized, double-blind, placebo-controlled trial evaluating the effect of Nesina (alogliptin) on the incidence of major adverse cardiovascular events in patients with type 2 diabetes mellitus. The primary objective of the trial is to establ
A 52-week, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of alogliptin when added on to metformin in pediatric patients ages 10 to 17 years (inclusive) with type 2 diabetes mellitus. At least 30% of rand
The trial has not begun but does not meet the criterion for delayed.
A prospective, randomized, controlled clinical trial that will assess the safety and efficacy of repetitive use of Cysview in the detection of bladder cancer.
Final protocol revisions submitted May 13, 2015. The study is anticipated to be initiated in fourth quarter of 2015
Deferred submission of pediatric studies for the long term maintenance treatment of asthma in patients 6 to less than 12 years of age until December 31, 2007.
Final study report submitted on 7/28/16.
Symbicort® pMDI (Budesonide and Formoterol Fumarate)
A long-term prospective observational cohort study (registry) of patients with Cushing's disease treated with Signifor (pasireotide[...]) to evaluate known and potential serious risks related to the use of Signifor (pasireotide [...]), includin
An assessment and analysis of spontaneous reports of serious (resulting in death, hospitalization, life-threatening, or disability) hyperglycemia, acute liver injury, and adrenal insufficiency in patients with Cushing's disease treated with Sig
A multi-center, randomized, clinical trial investigating the management of Signifor (pasireotide [...])-induced hyperglycemia in patients with Cushing's disease treated with Signifor (pasireotide[...]).
Conduct a trial in the >/=12 - 16 year old age group to evaluate the dose effect of phenylephrine hydrochloride injection on blood pressure in patients undergoing general anesthesia and neuroaxial anesthesia. Administration by both the bolus an
"The draft protocol was submitted[...] on December 18, 2013 with a cross reference letter to the NDA and to comply with the postmarketing requirement protocol submission. FDA responded indicating that the proposed study is on Full Clinical Hold
A prospective, multi-center, long-term, observational, registry study, of short bowel syndrome patients treated with teduglutide in a routine clinical setting, to assess the long-term safety of teduglutide. Design the study around a testable hy
An assessment and analysis of spontaneous reports of malignancy, teratogenicity, and hepatic abnormalities in patients treated with Juxtapid (lomitapide) for a period of 10 years from the date of approval. Specialized follow-up should be obtain
A long-term prospective observational study (product exposure registry) of patients with homozygous familial hypercholesterolemia (HoFH) treated with Juxtapid (lomitapide) to evaluate known and potential serious risks related to the use of Juxt
The first interim report submission milestone was missed because the applicant wishes to harmonize reporting dates to FDA and EMA. FDA determined that the applicant demonstrated “good cause” for the delay.
Open label, single dose pharmacokinetic study of Zecuity (sumatriptan) iontophoretic transdermal system in adolescents 12 to 17 years of age with a history of migraine, which compares the results with appropriate adult historical control data.
Per FDA letter dated 10/28/2016, this PMR has been fulfilled.
TEVA BRANDED PHARMACEUTICAL PRODUCTS R AND D INC
Randomized, double-blind, placebo-controlled, parallel group study to evaluate the effectiveness and safety of a single Zecuity (sumatriptan) iontophoretic transdermal system compared to a single placebo iontophoretic transdermal system in adol
The final protocol submission milestone was missed because we were waiting for data from the PK study (PMR 2000-1) and had not yet agreed on study design; pediatric studies are now on hold while sponsor investigates safety issues.
Open label, 12-month study to evaluate the long-term safety of Zecuity in adolescents 12 to 17 years of age with a history of migraine. Safety assessments must include adverse events, subject and investigator skin irritation evaluations, and mo
Deferred pediatric study under PREA: A pharmacokinetic, efficacy, and safety study of Nucynta ER for the management of chronic pain in pediatric patients ages 7 to <17 years.
Clinical trial (existing trial or new clinical trial) in which at least 30 patients are studied. The following examinations should be performed in these patients at baseline, 2 and 6 weeks after drug administration and 2-8 weeks after discontin
Agency issued communication on 14-OCT-14 that the FSR related to PMR did not fulfill PMR requirements. Pfizer proposed remediation plans and revised milestones which were acknowledged by FDA 8-DEC-14. Target accrual would be 30 pts who complete
Conduct a multiple dose trial in patients to determine how to adjust the crizotinib dose when it is coadministered with a strong CYP3A inhibitor (e.g., ketoconazole).
On 18-FEB-14, Pfizer submitted a revised timeline and study design change to the FDA to use “itraconazole” instead of ketoconazole due to revised safety guidelines. New milestones acknowledged 10-APR-14
Conduct a multiple dose trial in patients to determine how to adjust the crizotinib dose when it is coadministered with a strong CYP3A inducer (e.g., rifampin).
Conduct a multiple dose trial to determine the appropriate crizotinib dose in patients with various degrees of hepatic impairment.
Status update on study was provided on 26-MAR-15 that included current issues with trial enrollment, a proposal and updated timeline. Revised timelines acknowledged 20-APR-15
To assess the adequacy of the current cut-off, conduct a clinical trial to explore response to crizotinib in ALK-negative patients based on current assay cut-off. This should be compared to historic controls and to the response in ALK-positive
To conduct exposure-response analysis for progression free survival, response rate, overall survival and safety endpoints utilizing data from confirmatory trial A8081014 and to submit the analysis plan for review.
An evaluation of the potential carcinogenicity of colchicine in a 2-year bioassay in rat.
An evaluation of the potential carcinogenicity of colchicine in either a 2-year bioassay or 6-month transgenic study in an appropriate mouse model.
A randomized controlled trial to evaluate the effect of long term rifaximin treatment on the gut flora. In vitro susceptibility testing to rifaximin and other antimicrobial drugs must be included.
Final report submission was due 12/31/14
A randomized, controlled clinical trial to evaluate whether lactulose is required in combination with rifaximin to delay time to onset of episode of overt hepatic encephalopathy.
Deferred pediatric study under PREA in children 12 years to 16 years and 11 months of age with Juvenile Rheumatoid Arthritis (JRA) A safety and population pharmacokinetic (PK) study in adolescents with JRA who are ages 12 years to 16 years and
The final study report was submitted on 12/15/15. The prior approval supplement was submitted on 6/7/16
Vimovo® (Esomeprazole Magnesium and Naproxen)
Conduct a long-term safety study of adolescents ages =13 years to 17 years with moderate to severe symptoms of primary Restless Legs Syndrome. The study must provide a descriptive analysis of safety data in pediatric patients during at least 12
Conduct a driving study in adolescent patients of legal driving age who have Restless Legs Syndrome, using diphenhydramine as active control.
A simulated driving trial in healthy adult subjects treated with an appropriate dose of gabapentin enacarbil determined in PMC 1588-12 that includes active comparator and placebo arms.
Conduct a randomized, placebo-controlled, double-blind, parallel-group clinical trial of gabapentin enacarbil at 300 mg/day, 450 mg/day and 600 mg/day in patients with moderate to severe symptoms of RLS.
A randomized, double-blind, placebo-controlled trial evaluating the effect of Tradjenta (linagliptin) tablets on the incidence of major adverse cardiovascular events in patients with type 2 diabetes mellitus. The primary objective of this trial
The final protocol milestone was missed, because the FDA was engaged in discussion with the applicant regarding the study design at the time the final protocol submission milestone was due and FDA determined that the applicant  demonstrated goo
A randomized, double-blind study in pediatric patients ages 5 to 17 years using an age-appropriate formulation for the maintenance of remission of ulcerative colitis.
The final protocol submission milestone was missed due to issues with the study and comparator drug. The sponsor provided sufficient justification for missing the final protocol milestone.
Conduct a study to assess the safety of Vituz (hydrocodone and chlorpheniramine) in approximately 400-450 children ages 6-17 years with symptoms of the common cold. The study can be conducted with a formulation containing hydrocodone, chlorphen
Vituz® (Hydrocodone Bitartrate and Chlorpheniramine Maleate)
An adequately powered safety and efficacy study in pregnant adolescent girls, 12 to 17 years of age, with nausea and vomiting of pregnancy who are appropriate candidates for pharmacologic therapy.
As of April 1, 2016, 12 investigators with 15 sites in total have enrolled 65 subjects of whom, 47 have completed the study and 7 were discontinued/early termination. Currently, there are 8 investigators with 10 active sites. One thousand eight
Diclegis® (Doxylamine Succinate and Pyridoxine Hydrochloride)
A juvenile neurotoxicity study in neonatal rats intended to assess the potential for Nuedexta to induce apoptotic neuronal degeneration in the human fetus. Dextromethorphan/quinidine should be administered during the postnatal period demonstrat
On March 26, 2014, the FDA issued a letter to the Sponsor requesting the submission of an additional study in order to fulfill the PMR.
Nuedexta® (Dextromethorphan Hydrobromide and Quinidine Sulfate)
Deferred submission of your pediatric studies for the treatment of invasive aspergillosis and serious fungal infections caused by Scedosporium apiospermum and Fusarium spp., including Fusarium solani, in patients intolerant of, or refractory to
The Sponsor submitted the final report on 12/18/2015
Conduct a multiple dose trial to determine the appropriate regorafenib dose in patients with severe renal impairment. Submit the final protocol for FDA review before conducting the trial.
A multiple ascending dose clinical trial in healthy adult volunteers to determine the maximum tolerated dose of tramadol and to inform the dosing for a thorough QT trial of tramadol.
Ultracet® (Acetaminophen and Tramadol Hydrochloride)
A clinical trial in healthy adult volunteers to assess the risk of QT prolongation with tramadol, i.e., a thorough QT (tQT) trial. This trial will provide information on cardiac depolarization and conduction effects of tramadol at therapeutic a
Trial completion milestone missed, FDA determined there was good cause for the delay
Convene a panel of experts in oncology and neurology to obtain recommendations regarding which patients, if any, who were excluded from the randomized clinical trial because of increased risk of seizure should be evaluated in a postmarketing sa
To perform and submit the trial, presently under SPA agreement, TTX404 "A Phase 3, Multicenter, Randomized Study to Evaluate the Substitution of Marqibo (Vincristine Sulfate Liposomes Injection, VSLI) Standard Vincristine Sulfate Injection (VSI
Sponsor discontinued enrollment in the PMR study February 6, 2015. A meeting with the division was held January 6, 2016 to discuss a possible alternative study. A proposed protocol was submitted and not found to be acceptable. Sponsor is in the
Due to the complexity of on site preparation of the final drug product, to study and report at six month intervals on the experience of health care practitioners (HCPs) regarding safety or technical problems with preparation of Marqibo in pract
The sixth interim report was submitted October 26, 2016. The sixth interim report was due 12/2015. The reports are delayed due to late launch of Marqibo. (Milestones were revised 10/24/2014)
Considering the relative complexity of preparation of Marqibo, to explore methods to simplify preparation of the final drug product, including the possibility of developing a formulation of liposomal encapsulation such that the step of heating
A prospective, randomized, controlled, double-blind, efficacy and safety study of FYCOMPA (perampanel) in children ages 2 years to < 12 years for the adjunctive treatment of partial onset seizures with a long term safety extension. The primary
Per FDA letter dated 07/27/2016, this PMR has been released.
Deferred pediatric study under PREA: A prospective, randomized, controlled, double-blind, efficacy and safety study of FYCOMPA (perampanel) for the adjunctive treatment of partial onset seizures in children ages 1 month to < 4 years with a long
A prospective, multiple dose, randomized, controlled, double-blind, safety and efficacy trial of FYCOMPA (perampanel) as adjunctive treatment of partial onset seizures when high doses of Fycompa are added to concomitant treatments in adults on
The final protocol milestone was missed due to the time required for the Agency and the applicant to come to agreement on the protocols for these trials. The FDA determined that the applicant demonstrated good cause for the delay. Acknowledge R
A prospective human physical dependence trial in patients. The subjects should be titrated to the approved therapeutic dose of FYCOMPA (perampanel) of 8-12 mg, and maintained at this dose for an appropriate amount of time. At the end of the tre
Conduct an observational multi-site inception cohort study of Pomalyst (pomalidomide) users to address the questions detailed below: 1. To determine the failure rate for each of the different types of initial VTE prophylaxis for multiple myelom
Conduct a clinical trial, per FDA guidance [Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosing and Labeling], in patients with baseline hepatic impairment to determine the influence of
Conduct a clinical trial, per FDA guidance [Pharmacokinetics in Patients with Impaired Renal Function--Study Design, Data Analysis, and Impact on Dosing and labeling, in patients with baseline renal impairment and those on chronic dialysis], to
A long-term observational safety study of at least 10 years duration comparing patients with HIV-associated lipodystrophy and excess abdominal fat treated with Egrifta compared to a similar group of patients not treated with Egrifta to assess p
The protocol finalization milestone was missed because of discussion with FDA regarding study design.
A prospective, randomized, placebo-controlled clinical trial to evaluate if Egrifta increases the risk of development or progression of diabetic retinopathy when administered to HIV-infected patients with lipodystrophy and concomitant diabetes.
The final report submission milestone was missed because of subject recruitment difficulties.
A randomized, double-blind, 26-week, active-controlled clinical trial comparing Dulera (mometasone furoate and formoterol fumarate) Inhalation Aerosol and mometasone furoate to evaluate the risk of serious asthma outcomes (hospitalizations, int
Dulera® (Formoterol Fumarate and Mometasone Furoate)
Conduct a pediatric pharmacokinetic, safety, and antiviral activity trial of Stribild with activity based on the results of HIV-1 RNA virologic response and safety monitoring over at least 48 weeks of dosing in pediatric subjects from 12 to < 1
The final study report submitted with efficacy supplement on 07/28/16 and under FDA review.
Stribild® (Elvitegravir, Cobicistat, Emtricitabine, and Tenofovir Disoproxil Fumarate)
Conduct a pediatric pharmacokinetic, safety, and antiviral activity trial of Stribild with activity based on the results of HIV-1 RNA virologic response and safety monitoring over at least 48 weeks of dosing in pediatric subjects from 6 to < 12
A request for release from this study was denied per 04/05/2016 FDA letter, but will reevaluate as another pediatric development program progresses.
An open-label pilot study assessing the efficacy and tolerability of Suclear in pediatric patients 12-16 years of age, inclusive. This study will nclude PK assessments.
The final study report milestone was missed. The sponsor requested withdrawal of NDA 203595 on 7/9/15 and does not intend to perform the required postmarketing studies.
Suclear™ (Sodium Sulfate, Potassium Sulfate, and Magnesium Sulfate; and PEG-3350, Sodium Chloride, Sodium Bicarbonate, and Potassium Chloride)
A randomized, single-blind, multicenter, dose-ranging study comparing the safety and efficacy of Suclear (up to 3 doses) versus community standard of care in pediatric patients 12-16 years of age, inclusive.
The final protocol submission milestone was missed. The sponsor requested withdrawal of NDA 203595 on 7/9/15 and does not intend to perform the required postmarketing studies.
A randomized, single-blind, multicenter, dose-ranging study comparing the safety and efficacy of Suclear (up to 3 doses) versus community standard of care in pediatric patients 3-11 years of age, inclusive.
The study has not begun but does not meet the criterion for delayed. The sponsor requested withdrawal of NDA 203595 on 7/9/15 and does not intend to perform the required postmarketing studies.
A randomized, single-blind, multicenter, dose-ranging study comparing the safety and efficacy of Suclear (up to 3 doses) versus community standard of care in pediatric patients 1-2 years of age, inclusive.
Assess the systemic exposure and pharmacokinetics of PEG 3350,[...] following oral administration of Suclear in an adequate number of pediatric patients, encompassing all relevant age groups. These assessments may be conducted as part of the PR
Assess the systemic exposure and pharmacokinetics of PEG3350, [...] following oral administration of Suclear to adult patients. These assessments may be conducted as part of 1998-6 (above).
The final protocol submission milestone was missed. The sponsor requested withdrawal of NDA 203595 on 7/9/15 and does not intend to perform the required postmarketing studies.
Deferred pediatric study under PREA, a randomized, double-blind, placebo controlled study to evaluate the efficacy, pharmacokinetics, and safety of pregabalin in pediatric patients with partial onset seizures ages 1 month through 3 years, inclu
Deferred pediatric studies under PREA for treatment of partial onset seizures in patients ages 1 month to 16 years. Milestone dates were revised to September 30, 2018.
Deferred pediatric study under PREA, a randomized, double-blind, placebo-controlled study to evaluate the efficacy, pharmacokinetics, and safety of pregabalin in pediatric patients with partial onset seizures ages 4 through 16 years, inclusive.
Deferred pediatric study under PREA, a 12 month open label extension study to evaluate the safety of pregabalin in pediatric patients with partial onset seizures ages 1 month through 16 years, inclusive.
Testicular toxicity trial to investigate the effects of ganciclovir on spermatogenesis in humans.
The original final study report due date of 12/31/2014 was extended to 3/31/2016 due to challenges in enrollment.
Conduct an open-label pilot study assessing the efficacy and tolerability of SUPREP in adolescents (12 years to 16 years). The adult formulation (and any age appropriate reformulations) will be evaluated for tolerability and efficacy in this pi
Per FDA letter dated 12/16/2016, this PMR has been fulfilled.
Conduct a randomized, single-blind, multicenter dose ranging study comparing the safety and efficacy of SUPREP to NuLytely in adolescents (12 years to 16 years).
The study completion milestone was missed due to safety and pharmacokinetic issues. Final report deferral extension granted per FDA letter dated 5/2/16.
Conduct a randomized, single-blind, multicenter dose ranging study comparing the safety and efficacy of SUPREP to NuLytely in children (3 years to 11 years).
Conduct a food effect clinical trial, per FDA guidance [Food-effect Bioavailability and Fed Bioequivalence Studies], in order to determine the effect of food on the pharmacokinetics of Pomalyst (pomalidomide). The trial should be conducted in p
Conduct a clinical trial, per FDA guidance [Drug Interaction Studies-Study Design, data Analysis, Implications for Dosing, and Labeling Recommendations], in order to determine the effects of a CYP1A2 inducer (such as montelukast) on the PK of P
A randomized, controlled clinical trial to evaluate the safety of rifaximin in patients with hepatic encephalopathy classified as Child-Pugh C, MELD > 19, and MELD > or = to 25 hepatic impairment.
The final protocol has not been submitted and the trial completion and final report submission milestone dates have been missed.
Conduct a prospective, randomized, placebo-control, double-blinded efficacy /safety trial of Potiga (ezogabine) for the treatment of partial-onset seizures in children aged 2 years to <12 years old.
A randomized, double-blind, 26-week, active-controlled clinical trial comparing Symbicort (budesonide and formoterol fumarate dihydrate) Inhalation Aerosol with budesonide HFA to evaluate the risk of serious asthma outcomes (hospitalizations, i
A randomized, double-blind, 26-week, active-controlled clinical trial comparing Advair Diskus (fluticasone propionate and salmeterol xinafoate inhalation powder) and fluticasone propionate inhalation powder to evaluate the risk of serious asthm
GLAXO GROUP LTD ENGLAND DBA GLAXOSMITHKLINE
Advair® Diskus® (Fluticasone Propionate and Salmeterol)
A randomized, double-blind, 26-week, active-controlled clinical trial comparing Advair Diskus (fluticasone propionate and salmeterol xinafoate inhalation powder) and Flovent Diskus (fluticasone propionate inhalation powder) to evaluate the risk
Submit the hypothesis for the final Overall Survival (OS) analysis from the required Clinical Trial (PMR), and the final OS analysis will be performed in accordance with the OS plan. The Study Completion refers to the Statistical Analysis Plan
To complete the ongoing clinical trial CRAD001M2302 entitled "A Randomized, Double-blind, Placebo-controlled Study of RAD001 in the Treatment of Angiomyolipoma in Patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiom
Assess possible cobicistat protease inhibitory activity in vivo by sequencing the protease in virologic failure subjects' isolates from Studies GS-US-236-0102, GSUS- 236-0103, GS-US-236-0121, GS-US-236-0123 and GS-US-236-0128.
Perform a clinical trial to better characterize the incidence of and risk factors for renal adverse events in women. Provide adequate renal monitoring in the proposed trial to assess renal safety employing a renal monitoring algorithm similar t
Conduct a pharmacokinetic (PK) sub-trial of the renal safety trial in women to evaluate the potential for a drug-drug interaction between Stribild and commonly used oral contraceptives. Intensive pharmacokinetic data on each oral contraceptive,
A Pharmacokinetics/Pharmacodynamics trial of Sorilux Foam, 0.005% under maximum use conditions in 20 evaluable pediatric subjects with plaque psoriasis of the scalp and body age 12 years to 16 years and 11 months. Evaluate the effect of the pro
16/20 subjects completed as of 10/6/2015. FDA Deferral Extension Granted letter signed 12/30/2014 changing Final Study report due date to: June 2017.
A PK/PD study to characterize aprepitant PK parameters following administration of a single dose of intravenous fosaprepitant, in combination with a 5HT3 antagonist and dexamethasone, in pediatric cancer patients ages 0 to 17 years undergoing t
Deferred pediatric study of pharmacokinetics and safety under PREA for the treatment of mild to moderately severe pain when the use of an opioid analgesic is appropriate in pediatric patients ages 2 to 17 years.
Deferred pediatric study of pharmacokinetics, safety and efficacy under PREA for the treatment of mild to moderately severe pain when the use of an opioid analgesic is appropriate in pediatric patients ages one month to 2 years.
Conduct and submit the results of a randomized withdrawal clinical trial to address longer-term efficacy for your drug at appropriate doses.
Sponsor requested withdrawal of the NDA. FDA acknowledged withdrawal request in letter dated 4/17/15.
Deferred, 1-year, pediatric efficacy and safety study under PREA for the treatment of type 2 diabetes in pediatric patients ages 10 to 17 years.
Final report was due November 2015. On October 14, 2015, FDA granted a pediatric deferral extension. The new final report submission date is November 2019. Firm reports 173 of 200 subjects enrolled as of October 1, 2016.
A 10-year, observational study to prospectively evaluate the incidence of fibrosing colonopathy in patients with cystic fibrosis treated with Zenpep (pancrelipase) Delayed-Release Capsules in the US and to assess potential risk factors for the
Shire commits to utilize an antibody screening cut point based on a mean + 1.645 standard deviation for assay values from treatment naïve Gaucher patients. Shire will utilize the same methodology to calculate the anti-imiglucerase ECL cut poin
Shire commits to revise the cut point for the confirmatory anti-velaglucerase and anti-imiglucerase screening assays to a level that is less than or equal to the cut point of the screening assay.
Shire commits to re-assess the IgE cut point for the current ECL methodology using a chemically synthesized hybrid control. Shire commits to support assay validation using patient baseline values.
Shire commits to develop an assay to measure the ability of patient antibodies to block the uptake of velaglucerase and imiglucerase into target cells.
A pharmacokinetic trial in patients with severe hepatic impairment (MELD 19 - 25 and MELD > 25). This may be performed as a sub study in the ongoing Phase 3 trial (RFHE3002, A multicenter, open label trial to evaluate the long term safety and t
A pharmacokinetic trial in patients with concurrent renal insufficiency and liver impairment to determine the extent of elevation of systemic exposure of rifaximin which may lead to worsening of hepatic function. The PK data should be collected
Deferred pediatric study of safety and pharmacokinetics (single- and multipledose) under PREA for the relief of moderate to severe pain in patients requiring a continuous, around-the-clock opioid treatment for an extended period of time in pedi
Original Final Report Due Date: 12/31/2014; Deferral Extension granted per FDA letter dated 06/24/2013.
Deferred study of efficacy, safety, and pharmacokinetics (single- and multipledose) under PREA for the relief of moderate to severe acute pain where the use of an opioid is appropriate in patients ages 0-2 years.
Deferral extension granted 6/24/2013. The study is ongoing as per revised milestone
Deferred study of safety and pharmacokinetics (single- and multiple-dose) under PREA for relief of moderate to severe acute pain where the use of an opioid is appropriate in patients ages 2-17 years.
A randomized, placebo-controlled, dose-finding study under PREA evaluating at least two doses of linagliptin as monotherapy in pediatric patients ages 10 to 16 years (inclusive).
The Final Report was submitted to FDA on August 31, 2016.
Deferred randomized and controlled pediatric study under PREA to evaluate efficacy, safety, and pharmacokinetics of linagliptin for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 16 years (inclusive) as monotherapy a
The protocol finalization milestone was missed because the final study design is dependent on completion of PMR 1766-1; the final study report for PMR 1766-1 is currently under FDA review. Original Final Report Due Date: September 30, 2017; Def
To develop, in consultation with CDRH, a validated test kit for PDGFR gene rearrangements for patients with MDS/MPD.
To develop, in consultation with CDRH, a validated test kit for D816V c-kit mutation in aggressive systemic mastocytosis.
Conduct and submit the results of a multicenter, randomized trial or trials to verify and describe the clinical benefit of nivolumab in combination with ipilimumab in previously untreated adult patients with unresectable or metastatic, BRAF V60
Multicenter, randomized, double-blind, parallel-group, bupivacaine- and placebo-controlled study to evaluate the safety, efficacy and pharmacokinetic profile of a single intraoperative administration of Exparel for postoperative analgesia in ad
Original Final Report Due Date: 02/28/2014; Deferral Extension granted per FDA letter dated 01/30/2014.
A multicenter, randomized, double-blind, parallel-group, Bupivacaine- and placebo-controlled study to evaluate the safety, efficacy and pharmacokinetic profile of a single intraoperative administration of Exparel for postoperative analgesia in
Original Final Report Due Date: 08/31/2015; Deferral Extension granted per FDA letter dated 08/12/2015.
A multicenter, randomized, double-blind, parallel-group, bupivacaine- and placebo-controlled study to evaluate the safety, efficacy and pharmacokinetic profile of a single intraoperative administration of Exparel for postoperative analgesia in
Original Final Report Due Date: 02/28/2017; Deferral Extension granted per FDA letter dated 12/29/2016.
This study is not scheduled to start until review of the study data for commitment 1834-3 is complete and an appropriate dose for this age group is identified
To develop a validated, sensitive, and accurate assay for the assessment of cellular uptake inhibition by cell surface mannose receptors due to the presence of neutralizing antibodies to ELELYSO (taliglucerase alfa) for injection that is expect
FDA issued a not fulfilled letter on 7/11/14. The final protocol milestone was missed. FDA determined that the applicant demonstrated “good cause” for the delay. A new final study report was submitted on 3/21/16.
To conduct an assessment of neutralizing anti-drug antibody (ADA) response and presence of antibodies against plant-specific sugars in ELELYSO (taliglucerase alfa) for injection in patient plasma samples. Validated assays (developed under 1895-
The final report submission was due 3/31/2014. Sponsor unable to complete this PMR until PMR 1895-2 is complete. FDA determined that the applicant demonstrated “good cause” for the delay.
To evaluate the long-term safety and efficacy of ELELYSO (taliglucerase alfa) for injection in a registry of Gaucher disease patients being treated with ELELYSO (taliglucerase alfa) for injection. Detailed clinical status information will be co
To evaluate the effect of ELELYSO (taliglucerase alfa) for injection on pregnancy and fetal outcomes, and to collect detailed clinical status information on newborns and infants whose mothers are treated with ELELYSO (taliglucerase alfa) for in
Conduct a trial in 100 evaluable pediatric patients with plaque psoriasis of the scalp and body ages 12 to 16 years, 11 months, to evaluate the safety and effect of Taclonex® (calcipotriene and betamethasone dipropionate) Topical Suspension, 0
Original Final Report Due Date: 10/31/2016.  Per FDA letter dated 01/22/2015, final report due date extended to 06/30/2017. Second Deferral Extension Granted per FDA letter dated 09/29/2015. Final report submission now due December 2017
Taclonex® (Calcipotriene and Betamethasone Dipropionate)
Deferred pediatric study under PREA for the treatment of major depressive disorder in pediatric patients aged 7 to 17. Conduct a study to obtain data on the efficacy and safety of vilazodone in the relevant pediatric population. This must be a
Schedule revised due to enrollment rate.
Deferred pediatric study under PREA for the treatment of major depressive disorder in pediatric patients aged 7 to 17. Conduct a second study to obtain data on the efficacy and safety of vilazodone in the relevant pediatric population. This mus
Original Final Report Due Date: 01/31/2016; Deferral Extension granted per FDA letter dated 08/28/2015.
A descriptive observational cohort study of insertion, localization, and removal related events associated with Nexplanon use. The proposed protocol should include details on the study design, methodology, and analysis plan. This study will nee
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To complete the clinical trial of hydroxyprogesterone caproate in women with a singleton pregnancy who had a previous spontaneous preterm birth (Protocol #17P-ES-003).
To complete the clinical follow-up study (Protocol #17P-FU-004) of children born to women who participated in Protocol #17P-ES-003.
Conduct an open-label, single dose, single arm, tolerability, PK/PD and safety study of dabigatran etexilate given at the end of standard anticoagulant therapy in children aged less than 1 year old.
BIPI submitted the final report for study 1160.105 to NDA 022512 on August 1, 2016. BIPI considers PMR 2139-1 to be fulfilled.
A clinical trial to assess the safety, efficacy, and pharmacokinetics of Ravicti (glycerol phenylbutyrate) and its metabolites (PBA, PAA and PAGN) during Ravicti (glycerol phenylbutyrate) treatment in pediatric patients with Urea Cycle Disorder
A randomized, controlled clinical trial to assess the safety and efficacy of Ravicti (glycerol phenylbutyrate) in patients with Urea Cycle Disorders who are treatment naïve to phenylbutyrate.
The final protocol has not been submitted and the trial completion milestone date has been missed.
A prospective study in the United States which includes the five year period of time after introduction of Cayston (aztreonam for inhalation) to the market to determine if decreased susceptibility to aztreonam is increasing in Pseudomonas aerug
Conduct a prospective, randomized, placebo-control, double-blinded efficacy/safety trial of Potiga (ezogabine) for the treatment of partial-onset seizures in children aged 1 month to <2 years old. This study must also utilize endpoints and asse
A five-year prospective epidemiological study using a large healthcare claims database to determine the incidence of thyroid cancer among patients with type 2 diabetes exposed to Victoza (liraglutide [rDNA origin]) Injection and patients with t
The Final Report Submission milestone was missed because additional analyses will be required based on discussion with FDA, and FDA determined that the applicant demonstrated “good cause” for the delay.
A medullary thyroid carcinoma case series registry of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of Vict
To conduct a clinical trial that assesses the agreement between Da Tscan imaging results and diagnostic outcomes among non-Caucasian and Caucasian patients. This trial will be designed and conducted in a manner that allows a comparison of the r
Final Protocol submitted after milestone date
A Randomized, Multicenter, Double Masked, Parallel-Group Study Assessing Safety and Efficacy of Loteprednol Etabonate Ophthalmic Gel, 0.5% versus Prednisolone Acetate Ophthalmic Suspension, 1% for the Treatment of Intraocular Inflammation Follo
Deferred pediatric study under PREA to assess the pharmacokinetics, safety, and tolerability in pediatric patients 6 to 16 years of age with minor soft tissue injuries.
A deferred pediatric study under PREA for the acute treatment of agitation associated with schizophrenia or bipolar I disorder in pediatric patients ages 10 to 17 years. A study to obtain pharmacokinetic data and provide information pertinent t
Per FDA letter dated 07/26/2016, this PMR has been fulfilled.
A deferred pediatric study under PREA for the acute treatment of agitation associated with schizophrenia or bipolar I disorder in pediatric patients ages 10 to 17 years. A study of the efficacy and safety of Adasuve (loxapine) inhalation powder
On November 18, 2013, Teva submitted extension request in Sequence 0074. On November 27, 2013, FDA granted Teva’s extension request for PMR 1891-2 with a final report submission date of April 13, 2018.
Conduct a large, non-randomized, open-label, postmarketing observational study to assess the risks of bronchospasm and related respiratory adverse events and serious outcomes (e.g., hospitalization, intubation, mechanical ventilation, or rescue
On August 26, 2015 (Sequence 0103), Teva requested a change in timetables for PMR 1891-4 study completion and final report submission due to the extremely slow patient enrollment into the requirement postmarketing observational study that was d
A clinical trial to assess the effect of mild, moderate and severe renal impairment on the pharmacokinetics of phentermine.
The final report submission milestone was missed and FDA determined that the applicant failed to demonstrate “good cause” for the delay.
A juvenile rat toxicology study. The study should utilize animals of an age range and stage(s) of development that are comparable to the intended pediatric population; the duration of dosing should cover the intended length of treatment in the
A large, long-term, prospective observational study in adult patients with relapsing multiple sclerosis, with the primary objective of determining the nature and incidence of serious infections including opportunistic infections, leiomyomata, m
Conduct a clinical trial to determine the pharmacokinetics of romidepsin in advanced cancer patients with moderate and severe hepatic impairment. Submit the protocol for agency review prior to commencing the trial.
Original Trial Completion due date was August 31, 2014. Previously revised milestones to trial completion: Aug 31, 2016 and Final Report due Aug 31, 2017. Nov 15, 2016, applicant requested new revised milestones. Based on original milestone dat
A pediatric study to evaluate pharmacokinetics (PK), efficacy for symptomatic relief of non-infectious diarrhea, and safety with different doses of Fulyzaq (crofelemer) over a four week period in HIV-positive pediatric patients, ages 1 month to
Final Protocol was due June 2013. Sponsor will submit in March 2014
A two-year rodent carcinogenicity study of orally administered crofelemer in the rat. The carcinogenicity protocol will be submitted for Special Protocol Assessment (SPA) prior to initiating the study.
An in vivo study in human subjects to evaluate whether crofelemer inhibits CYP 3A4 using a probe that is a pure substrate of CYP 3A4.
Final Protocol not yet submitted. Sponsor will submit in March 2014
Deferred pediatric study under PREA for the treatment of hyperphosphatemia and chronic kidney disease on dialysis in pediatric patients ages 0-18.
Per FDA letter dated 12/14/2016, this PMR has been fulfilled
Conduct a study to evaluate the effects of Impavido (miltefosine) on spermatogenesis and male hormones in patients with leishmaniasis receiving Impavido (miltefosine) treatment. Evaluations will include semen volume, sperm count, sperm concentr
Conduct a dedicated QT study in leishmaniasis patients receiving Impavido (miltefosine) treatment to evaluate the effects of Impavido (miltefosine) on the QT interval. ECGs and PK samples will be obtained to identify potential effects of Impavi
Conduct a descriptive study regarding efficacy outcome and adverse reactions in patients with leishmaniasis who weigh more than 75kg.
A milk-only lactation trial in lactating women receiving Linzess (linaclotide) therapeutically to assess concentrations of linaclotide and its active metabolite in breast milk using a validated assay in order to appropriately inform the Nursing
A prospective, randomized, controlled clinical trial that will assess the safety and efficacy of Cysview in the detection of carcinoma in situ of the bladder.
Deferred pediatric study of pharmacokinetics and safety under PREA for the treatment of moderate to severe pain where an opioid analgesic is appropriate in pediatric patients ages 2 to 17 years.
The final report milestone was not missed, however, the sponsor did not achieve study endpoints for PK and subsequently received a complete response
Deferred pediatric study of pharmacokinetics, safety and efficacy under PREA for the treatment of moderate to severe pain where an opioid analgesic is appropriate in pediatric patients ages birth to 2 years.
The final report milestone was missed, because of ongoing study design discussions with the Division
Deferred pediatric study under PREA for the treatment of overactive bladder in the subgroup of pediatric patients with neurologic disease ages 6 to 16 years, 11 months.
Study A0221047 has been initiated with pediatric patients being screened and enrolled. Final Study report due on 6/30/17
A single- and repeated-dose pharmacodynamics and pharmacokinetics trial of Giazo tablets administered orally to pediatric patients ages 12 years to less than 17 years with mildly to moderately active ulcerative colitis to support pediatric labe
The final protocol submission milestone and the final study report milestones were missed.
A placebo-controlled clinical trial in female patients with active ulcerative colitis to assess the efficacy of an eight week course of Giazo therapy for the treatment of active disease in this patient population.
A pharmacokinetic trial in patients to evaluate the effect of concomitant therapy with antibiotics commonly used in ulcerative colitis on the metabolism of balsalazide following administration of Giazo.
A dose-ranging clinical trial to evaluate the risk-benefit ratio of Lusedra in patients classified as ASA III or IV, adult patients weighing less than 60 kg, and geriatric patients.
A study in adolescents and younger pediatric patients receiving emetogenic chemotherapy (HEC or MEC) to evaluate fosaprepitant PK, safety, and tolerability.
A drug utilization study to better characterize the reporting rates of adverse events associated with the long-term use of Korlym (mifepristone). These data will provide a denominator for the adverse events of special interest (endometrial hype
A drug-drug interaction clinical trial to determine a quantitative estimate of the change in exposure of mifepristone following co-administration of ketoconazole (a strong CYP3A4 inhibitor).
The original milestone for the final protocol submission (August 2012) was missed. Final reports for two previously conducted trials were submitted by the applicant on August 24, 2012. FDA issued a PMR Not Fulfilled letter on May 3, 2013. The a
A 10 year, observational study to prospectively evaluate the incidence of fibrosing colonopathy in patients with cystic fibrosis treated with Ultresa (pancrelipase) Delayed-Release Capsules in the U.S. and to assess potential risk factors for t
An observational study to estimate the prevalence of antibody seropositivity to selected porcine viruses in patients taking Ultresa (pancrelipase) Delayed-Release Capsules compared with an appropriate control group.
Approximately 1013 subjects have been enrolled out of a planned 1300 subjects. The Study completion milestone and the final study report milestone was missed, because of additional time needed to enroll a larger number of subjects than original
Phase 2 open-label trial to evaluate the safety, efficacy and pharmacokinetics of peginesatide injection for maintenance treatment of anemia in pediatric subjects with chronic kidney disease (CKD) on hemodialysis who are currently stable on ESA
NDA product withdrawn from the market during investigation of adverse events. Studies on hold.
Phase 2 open-label follow-up trial to evaluate the safety, tolerability, and efficacy of peginesatide injection for maintenance treatment of anemia in pediatric subjects with CKD on hemodialysis.
NDA product withdrawn from the market during investigation of adverse events. Studies on hold
Phase 3 randomized, active-controlled, open-label, multicenter trial to evaluate the efficacy and safety of peginesatide injection for maintenance treatment of anemia in pediatric subjects with CKD on dialysis.
Phase 3 open-label follow-up extension trial to evaluate the safety, tolerability and efficacy of peginesatide injection for maintenance treatment of anemia in pediatric subjects with CKD on dialysis.
Post marketing comparative observational safety study of dialysis patients (both incident and prevalent) receiving Omontys versus a U.S. marketed ESA to assess safety of long term use. Provide the protocol and analysis plan for FDA review and c
NDA product withdrawn from the market during investigation of adverse events. Studies on hold. Good Cause Letter sent May 3, 2013.
Conduct a prospective randomized, controlled trial (RCT) of Omontys versus a U.S. marketed ESA in anemic patients with chronic kidney disease (CKD) who are in the time interval around the initiation of dialysis (defined as incident dialysis pat
NDA product withdrawn from the market during investigation of adverse events. Studies on hold. Good Cause Letter sent May 3, 2013
To conduct a study to determine whether varenicline, as part of an overall smoking cessation program, is effective in achieving and maintaining smoking cessation in tobacco-addicted adolescents, ages 12 through 16, inclusive, to determine a saf
Study enrollment was initiated in April 2011. On March 25, 2015 Pfizer submitted a request to extend the due date for the final study report to December 6, 2018, due to difficulties with enrollment of the younger adolescent cohort. A revised PW
To conduct a prospective epidemiologic cohort study in pregnant women who are smokers and who are exposed to varenicline at the time of conception or any time during pregnancy. This information will be used to assess the potential risk to the f
Conduct an extension of the randomized, double-blind, active- and placebo controlled trial to compare the risk of clinically significant neuropsychiatric events in individuals using Chantix (varenicline), bupropion, nicotine replacement therapy
Deferred pediatric study under PREA, a pharmacokinetic and safety study for the treatment of moderate to severe chronic pain requiring continuous, around-the clock opioid treatment for an extended period of time in pediatric patients ages 7 thr
Sponsor has not provided any update. A Deferral was granted on 3/2/2015, with a final report submission in Dec. 2016.
Conduct an open-label trial to evaluate the pharmacokinetics, safety and tolerability of nintedanib in patients with hepatic impairment (Child-Pugh Classification A and B) compared to healthy subjects
Original Final Report Due Date: 01/31/2015; Deferral Extension granted per FDA letter dated 05/17/2016.
Pharmacokinetic, safety, and efficacy study in subjects from birth to 2 years of age.
Deferred pediatric study under PREA for the treatment of major depressive disorder in pediatric patients aged 7 to 17. Conduct a study to obtain data on the efficacy and safety of desvenlafaxine in the relevant pediatric population.
Submit the results of the protocol-specified final analysis of overall survival, along with datasets and analysis SAS programs, from Study 14874, A randomized, double-blind, placebo-controlled phase III study of regorafenib plus best supportive
Subpart I Confirmatory Studies: We note your commitment to provide, within 1 month of the date of this letter, detailed protocols to address the requirement to conduct postmarketing studies, such as field studies, to verify and describe pyridos
UNITED STATES ARMY OFFICE SURGEON GENERAL
A randomized dose-comparison trial in patients with progressive metastatic medullary thyroid cancer comparing the safety and activity of oral cabozantinib 140 mg daily to a biologically active and potentially safer lower daily cabozantinib dose
Submit the results of the protocol-specified final analysis of overall survival, along with datasets and analysis programs, from Protocol XL184-301.
Conduct a pediatric safety and antiviral activity study of rilpivirine with activity based on the results of virologic response over at least 48 weeks of dosing and safety monitored over 48 weeks in pediatric subjects from 4 weeks to <12 years
The final protocol due date of 06/20/2013 has been already passed and the Sponsor submitted only the partial protocol.
You must conduct a PK trial in children 6 to <12 who may benefit from the drug (i.e., not in otherwise healthy pediatric volunteers). You should conduct a single and multiple dose PK trial that would characterize ibuprofen and phenylephrine pro
Per FDA letter dated 06/28/2016, this PMR has been fulfilled.
Advil® Congestion Relief (Ibuprofen and Phenylephrine Hydrochloride)
You must conduct a PK trial in children 2 to <6 who may benefit from the drug (i.e., not in otherwise healthy pediatric volunteers). You should conduct a single and multiple dose PK trial that would characterize ibuprofen and phenylephrine prof
Per FDA letter dated 07/11/2016, this PMR has been released.
You must conduct a PK trial in children 6 to <12 who may benefit from the drug (i.e., not in otherwise healthy pediatric volunteers). You should conduct a single and multiple dose PK trial that would characterize ibuprofen, phenylephrine, and c
Advil® Allergy and Congestion Relief (Ibuprofen, Phenylephrine Hydrochloride, and Chlorpheniraline Maleate)
You must conduct a PK trial in children 2 to <6 who may benefit from the drug (i.e., not in otherwise healthy pediatric volunteers). You should conduct a single and multiple dose PK trial that would characterize ibuprofen, phenylephrine, and ch
Deferred pediatric studies under PREA for the cardiac and oncology indications in pediatric patients ages 1 year to 16 years old.
Per FDA letter dated 05/16/2016, this PMR has been fulfilled.
Deferred pediatric study under PREA for the treatment of hypertension in pediatric patients ages 1 to 18.
Deferred pediatric study under PREA for the treatment of Major Depressive Disorder in pediatric patients ages 7 to 17 years. A study of safety and efficacy of Oleptro (trazodone hydrochloride) extended release tablets in the relevant pediatric
Randomized, double-blind, dose-controlled clinical trial of fospropofol disodium injection in neonates (less than one month of age) undergoing sedation for procedures such as lumbar puncture, MRI, and/or circumcision. Pharmacokinetics will be s
A randomized, single-blind, multicenter dose-ranging study to obtain pharmacokinetic data and to compare the safety and efficacy of HalfLytely and Bisacodyl Tablet versus NuLYTELY in children (6 - 11 years of age).
Deferred study under PREA to evaluate the pharmacokinetic, pharmacodynamic, and safety profiles of dexlansoprazole in patients 1 month to 11 months of age with endoscopy-proven erosive esophagitis (EE).
The study has not begun but does not meet the criterion for delayed. Deferral extension granted until 6/30/2019.
Deferred study under PREA to evaluate the pharmacokinetics, healing, maintenance of healing, and symptoms of endoscopy-proven erosive esophagitis (EE) in patients 1 year to 11 years of age.
Deferred study under PREA to evaluate the long-term safety of dexlansoprazole for the healing and maintenance of healing of erosive esophagitis (EE) in pediatric patients 1 month through 11 months of age, who require chronic treatment with dexl
Deferred study under PREA to evaluate the long-term safety of dexlansoprazole for the healing and maintenance of healing of erosive esophagitis (EE) in pediatric patients 1 year through 17 years of age, who require chronic treatment with dexlan
An epidemiological study to address whether this formulation of Oxycodone HCl results in a decrease in misuse and abuse, and their consequences: overdose, death and addiction.
PK/Efficacy/Safety study in pediatric subjects ages 2 years to 17 years 11 months with tinea corporis.
Per FDA letter dated 11/10/2016, this PMR has been fulfilled.
A 2-year dermal rat carcinogenicity study.
Per Agency instruction, this study was terminated on August 5, 2015, due to increased mortality.
Deferred pediatric study under PREA to evaluate the safety and pharmacokinetics of raltegravir in HIV-exposed neonates (born to HIV-infected mothers). This multiple-dose pharmacokinetic and safety study will evaluate raltegravir in addition to
The PREA Deferral Extension, requested on May 20, 2016, was granted on June 30, 2016 and the final report due date is revised from December 2016 to September 2017.
A randomized and controlled pediatric study under PREA to evaluate the safety, efficacy, and pharmacokinetics of BYDUREON (exenatide extended-release for injectable suspension) for the treatment of type 2 diabetes mellitus in pediatric patients
A 2-year study in mice to determine the reversibility of C-cell hyperplasia, the potential of hyperplasia to progress to neoplasia, and GLP-1 receptor expression on C-cells after 6 months of treatment with exenatide for injectable suspension.
A study to evaluate and compare GLP-1 receptor expression/density on human, rat, and mouse thyroid C-cells. This should include evaluation of mouse tissue from PMR 1860-2 following exenatide for injectable suspension treatment for 6 months as w
A study to evaluate the dependence of the GLP-1 receptor for exenatide-induced C-cell hyperplasia and investigate the expression of growth regulatory genes in wild-type and GLP-1 receptor knock-out mice.
A medullary thyroid carcinoma case series registry of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of BYDU
A randomized, double blind, placebo-controlled trial evaluating the effect of BYDUREON (exenatide extended-release for injectable suspension) on the incidence of major adverse cardiovascular events (MACE) in subjects with type 2 diabetes mellit
A randomized trial comparing pralatrexate in combination with systemic bexarotene versus systemic bexarotene alone in patients with cutaneous T-cell lymphoma (CTCL) who are refractory to at least one prior systemic therapy. Description of trial
Thirty evaluable patients are enrolled in the study and the study is ongoing. The Phase 1 trial completion date (August 31, 2011) and the Phase 3 Protocol submission date (September 30, 2011) have been missed.
A clinical pharmacokinetic trial in patients with renal impairment to include patients with severe renal impairment. Description of trial: This will be a Phase 1 clinical trial to evaluate the pharmacokinetics of pralatrexate in relapsed/refrac
Enrollment is continuing in all age groups.
Pharmacokinetic and safety study in subjects >2 years to <17 years of age.
To date, 100 patients have been screened, 79 enrolled, and 72 have completed the study
A study to determine patient compliance with dosing instructions in the setting of actual clinical use. The study should enroll patients representing the clinical population using the drug, and should assess the incidence, nature, causes, and c
Deferred pediatric study of tizanidine for acute treatment of spasticity in pediatric patients ages 0 to 16.
The final report milestone was missed. Deferral Extension Requested October 15, 2014. Denied per FDA letter dated March 3, 2015. Sponsor has not made good faith attempts to conduct the required studies. PREA noncompliance letter was issued Apri
Inclusion of a new target population, men with osteoporosis, in the required postmarketing study entitled, “The Denosumab Global Postmarketing Safety Observational Study"
Inclusion of a new target population, men with osteoporosis, in the required postmarketing study entitled, “The Prolia Postmarketing Active Safety Surveillance Program”
An open-label study to assess the effect of Enstilar® (calcipotriene and betamethasone dipropionate) Foam, 0.005%/0.064% on calcium metabolism in 100 evaluable pediatric subjects aged 12 years to 16 years and 11 months with plaque psoriasis of
To conduct an adequate and well-controlled long-term maintenance study to evaluate the efficacy and safety of asenapine in the treatment of adults with acute manic or mixed episodes associated with bipolar I disorder. The maintenance study shou
A randomized and controlled pediatric study under PREA to evaluate the efficacy and safety of liraglutide for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 16 years 11 months. This study must not be initiated until
174 patients have been screened; 73 have been randomized.
Deferred pediatric trial under PREA to evaluate the safety and efficacy of multiple doses of mometasone MDI in children 5 to 11 years of age with asthma.
The PMR was fulfilled per FDA letter dated 7/12/2016
A deferred pediatric study under PREA for the treatment of major depressive disorder in pediatric patients ages 12 to 17 years. Conduct a study to obtain data on the pharmacokinetics (PK), efficacy and safety of levomilnacipran in the relevant
Original Final Report Due Date: 07/25/2018; Deferral Extension granted per FDA letter dated 01/28/2016.
A deferred pediatric study under PREA for the treatment of major depressive disorder in pediatric patients ages 7 to 17 years. Conduct a study to obtain data on the efficacy and safety of levomilnacipran in the relevant pediatric population (ag
Original Final Report Due Date:07/25/2020; Deferral Extension granted per FDA letter dated 01/28/2016
To support the use of levomilnacipran in children less than 12 years of age, you must conduct a study to assess the safety of levomilnacipran in juvenile rats. This study must include evaluation of neurological/behavioral development and reprod
Test for the individual component phytosterol content in all batches of Clinolipid (lipid injectable emulsion, USP) 20%, manufactured over a three year period, using the method developed under PMR 2085-1. Based on these test results, establish
Develop and validate an appropriate analytical method for measuring phytosterol levels in plasma.
The final report was submitted on 10/31/2014. FDA determined not to fulfill the PMR because the validation parameters and the analytical method were incomplete per the letter dated 12/10/2015.
Randomized controlled trial to evaluate the risk of developing essential fatty acid deficiency (EFAD) in pediatric patients, including neonates, receiving either  Clinolipid (lipid injectable emulsion, USP) 20% or standard of care soybean oil b
Randomized controlled trial in pediatric patients, including neonates, comparing Clinolipid (lipid injectable emulsion, USP) 20% with a phytosterol-depleted formulation of Clinolipid (lipid injectable emulsion, USP) 20% and another standard-of-
Randomized clinical trial in hospitalized patients receiving either Clinolipid (lipid injectable emulsion, USP) 20% or other standard-of-care IV lipid emulsions to evaluate clinical safety outcomes of sepsis and mortality. In addition, the tria
A randomized, controlled, parallel group, superiority study in pediatric patients ages 10 through 17 years to evaluate the safety and efficacy of glatiramer acetate compared to an appropriate control for the treatment of relapsing forms of mult
Per FDA letter dated 06/07/2016, this PMR has been released.
A juvenile animal toxicology study in rats to evaluate the effects of glatiramer acetate on growth, reproductive development, and neurological and neurobehavioral development.
Deferred pediatric study under PREA for the treatment of major depressive disorder in pediatric patients aged 7 to 17. Conduct a study to obtain data on the efficacy and safety of desvenlafaxine in the relevant pediatric population.
Continue follow-up of patients (on treatment and in protocol defined posttreatment follow-up) enrolled in Study 200-WW at least an additional 2 years past the March 28, 2011 cut-off date. Submit the Final Report, which will consist of an update
Deferred pediatric study under PREA for the treatment of acute deep vein thrombosis when administered in conjunction with warfarin sodium in pediatric patients ages one to 16 years.
Deferred pediatric study under PREA. Final protocol submitted and accepted. No subjects have been enrolled in the study. Final report due December 15, 2019
Deferred pediatric study under PREA for the treatment of acute pulmonary embolism when administered in conjunction with warfarin sodium in pediatric patients ages one to 16 years.
Randomized clinical trial comparing Clinolipid (lipid injectable emulsion, USP) 20% to another standard-of-care IV lipid emulsion, evaluating long-term risk of developing essential fatty acid deficiency (EFAD) and parenteral nutrition associate
The final protocol milestone was missed due to challenges selecting study endpoints and duration. The sponsor is working with FDA on the protocol.
Based on the data obtained in PMR #1876-1 and PMR#1876-2, submit a toxicological risk assessment for all leachables in the drug product detected during stability. Your risk assessment should include data to justify the safety of the leachables
To commit to development and validation of an assay to detect the presence of neutralizing antibodies to Oncaspar. Validation studies will provide an assessment of the sensitivity (in mass units of antibodies), specificity, and reproducibility
Deferred study under PREA to evaluate the pharmacokinetics, pharmacodynamics, and safety of omeprazole in patients 1 month to 11 months of age with erosive esophagitis (EE).
Per FDA letter dated 02/03/2016, this PMR has been fulfilled.
Conduct an open-label, repeated treatment, pediatric study in 130 patients, 12-16 years of age with severe axillary hyperhidrosis that is inadequately managed with topical agents.
Final report submitted on 05/30/2008. Labeling supplement was submitted 09/29/2008.
Botox® and Botox® Cosmetic (OnabotulinumtoxinA)
Conduct an open-label, randomized, parallel-group, active-controlled, multicenter, non-inferiority efficacy study of dabigatran etexilate versus standard of care for venous thromboembolism treatment in children from birth to less than 18 years
A total of 40 of the planned 240 patients have been entered into this study. The study is ongoing.
Conduct an open label, single arm trial to evaluate safety of dabigatran etexilate for secondary prevention of venous thromboembolism in children aged 0 to less than 18 years. The anticipated enrollment is 100 patients. Submit the clinical stud
A total of 30 of the planned 100 patients have been entered into this study. The study is ongoing
Phase 2 trial in 50 pediatric patients 1month to 16 years of age to evaluate pharmacokinetics, pharmacodynamics, and safety data in patients with solid tumors without bone marrow involvement. Submit the protocol for Agency review and concurrenc
The trial completion milestone was June 2016. Recruitment efforts were slower than expected causing a delay in trial completion. Teva requested a revised timeline on March 31, 2016 which was agreed to May 11, 2016
To conduct an assessment for neutralizing antibodies using the validated assay developed under PMR 3 in all patients with binding antibodies to tbo-filgrastim or native G-CSF and in all patients with evidence of unexplained, persistent neutrope
Deferred requirement for development of an age appropriate formulation: Develop an age appropriate formulation to allow for dosing for ages 1 to <5 years.
Astellas is assessing a current body of data which supported the approval in Europe and other countries of a tacrolimus immediate- release granule formulation (Modigraf®) which may be appropriate for dosing pediatric patients < 5 years. Astell
PMR-EC-1206 A Phase II, Open-Label, Multi-Center Study to Compare the Pharmacokinetics of Tacrolimus in Stable Pediatric Allograft Recipients Converted from a Prograf® Based Immunosuppressive Regimen to a Tacrolimus Prolonged Release, Advagraf
A total of 81 pediatric patients have been exposed to tacrolimus extended release capsules as of the close of the reporting period. Subjects continue to be followed for 1 year. The last subject is expected to complete the 1-year follow-up in Oc
Identify an optimal dose of INJECTAFER (ferric carboxymaltose injection) for the pediatric patient population. Conduct one or more pharmacokinetic (PK) and pharmacodynamic (PD) trials in pediatric patients aged 1 to <17 years with iron deficien
The study completion date has passed and the study has not yet been completed.
Determine the safety and efficacy of INJECTAFER (ferric carboxymaltose injection) in pediatric patients aged 1 to <17 years with iron deficiency anemia by conducting a randomized, active-controlled clinical trial.
A randomized, controlled trial to rule out a moderate increase in the risk of serious cardiovascular events with tocilizumab, e.g., stroke, non-fatal MI, cardiovascular death.
An open-label pharmacokinetic and safety study or studies of an age appropriate formulation of indomethacin in pediatric patients 6 through 17 years of age
2 patients have been enrolled into the trial
An open-label pharmacokinetic and safety study or studies of an age appropriate formulation of indomethacin in pediatric patients 2 through 6 years of age
The study has not been initiated, but does not meet the criterion for delayed
A pharmacokinetic, safety, and efficacy study or studies of an age appropriate formulation of indomethacin in pediatric patients 1 through 2 years of age
Complete the trial and submit the final report of CNTO328MCD2002 “An Openlabel, Multicenter Study to Evaluate the Safety of Long-term Treatment with Siltuximab in Subjects with Multicentric Castleman’s Disease.”
A controlled efficacy and safety study of lurasidone in the treatment of pediatric patients (ages 10 to 17 years) with a diagnosis of depressive episode associated with bipolar disorder
Original Final Report Due Date: 12/30/2016; Deferral Extension granted per FDA letter dated 12/06/2016.
A long-term, open-label safety study of study of lurasidone in the treatment of pediatric patients (ages 10 to 17 years) with a diagnosis of depressive episode associated with bipolar disorder
PMCs in 6/28/13AP ltr. Status has not been reported yet.
A placebo-controlled, randomized withdrawal maintenance study of lurasidone in patients with bipolar I disorder
A clinical trial to assess the risk of QT prolongation with Zubsolv sublingual tablet, i.e., a thorough QT (tQT) trial.
Zubsolv® (Buprenorphine Hydrochloride and Naloxone Hydrochloride Dihydrate )
A 26-week randomized, double-blind, placebo controlled study of the safety, efficacy, and pharmacokinetics (PK) of Trulicity (dulaglutide) for the treatment of type 2 diabetes mellitus in pediatric patients ages 10 to 17 years (inclusive) with
A study to evaluate dulaglutide toxicity in immature rats.
Per FDA letter dated 02/23/2016, this PMR has been fulfilled.
A medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction o
The final protocol milestone was missed, because the FDA was engaged in discussion with the applicant regarding the study design at the time the final protocol submission milestone was due and FDA determined that the applicant demonstrated good
A 26-week randomized, controlled trial comparing once weekly Trulicity (dulaglutide), 0.75 mg and 1.5 mg, with insulin glargine on glycemic control in patients with type 2 diabetes mellitus and moderate or severe renal impairment, with a 26-wee
FDA has determined that you had good cause for your failure to comply with the Trial Completion milestone of the original timetable of 01/31/2017.
Conduct a trial to evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response) of SOVALDI (sofosbuvir) as a component of an antiviral treatment regimen in pediatric subjects 3 through 17 years of age with c
Reported enrollment of 62 subjects as of this reporting period.
Collect and analyze long-term safety data for subjects enrolled in the pediatric SOVALDI (sofosbuvir) pharmacokinetic, safety and efficacy trial described in 2110-1. Data collected should include at least 3 years of follow-up in order to charac
Reported 7 subjects as of this reporting period.
Establish a pregnancy registry of patients with RA who become pregnant while on anti-rheumatic therapy, including  Adalimumab as well as other medications, which will run for 3 years.
Enrollment completed on 8/22/14 (944 patients), 1-year follow-up ongoing. 8/22/14 Final Study Report due date revised to be submitted 9/2017
Submit the final report and datasets for the ongoing trial GS-US-334-0153, entitled, “A Phase 3B Randomized, Open-Label, Multi-Center Trial Assessing Sofosbuvir + Ribavirin for 16 or 24 Weeks and Sofosbuvir + Pegylated Interferon + Ribavirin
Submit the final report and datasets for the ongoing trial GS-US-334-0125, entitled, “A Phase 2, Multicenter, Open-Label, Randomized Study to Investigate the Safety and Efficacy of GS-7977 and Ribavirin Administered for 48 Weeks in Patients I
Submit an interim report from the ongoing study GS-US-248-0122, entitled, “A Long Term Follow-up Registry for Subjects Who Achieve a Sustained Virologic Response to Treatment in Gilead-Sponsored Trials in Subjects with Chronic Hepatitis C Inf
Genzyme commits to designing and implementing an infantile-onset Pompe disease study to assess growth and development with treatment with alglucosidase alfa, in patients who are less than one year of age at study entry.  This study is to includ
Conduct a pediatric trial to evaluate the pharmacokinetics, safety and antiviral activity (efficacy) of abacavir/dolutegravir/lamivudine FDC tablets in HIV infected pediatric subjects 2 years to less than 6 years of age. The safety and antivira
Conduct a pediatric trial to evaluate the pharmacokinetics, safety and antiviral activity (efficacy) of abacavir/dolutegravir/lamivudine FDC tablets in HIV infected pediatric subjects 6 years to less than 12 years of age and in children older t
Conduct a safety and efficacy study in pediatric patients with chronic idiopathic constipation ages ¡Ý 6 Years to <18 Years (SAG/0211PFC-1131)
New pediatric studies to replace 572-1. The sponsor reports that 606 subjects have been enrolled and enrollment is complete.
Conduct a safety and efficacy study in pediatric patients with chronic idiopathic constipation ages ¡Ý 6 Months to < 6 Years.
New pediatric studies to replace 572-1; the study has not been initiated, but does not meet the criterion for delayed
Collect, analyze, and submit data on subjects with cirrhosis including decompensated cirrhosis who achieve sustained virologic response following treatment with a sofosbuvir-based regimen to evaluate durability of virologic response and to char
Conduct a study of pretreatment molecular subtyping of tumors from patients treated in the postmarketing cardiac safety trial (PMR#2) and submit an exploratory analysis of the relationship of pathological complete response with the different tu
Conduct an open-label pharmacokinetics and safety study of Xartemis XR in pediatric patients ages 12 to less than 17 years with acute pain severe enough to require opioid treatment and for which alternative treatment options are inadequate.
Original Final Report Due Date: 03/31/2016; Deferral Extension granted per FDA letter dated 01/06/2017.
Xartemis™ XR (Acetaminophen and Oxycodone Hydrochloride)
Conduct an open-label pharmacokinetics and safety study of an age-appropriate formulation (oxycodone hydrochloride/acetaminophen) in pediatric patients ages 2 to less than12 years with acute pain severe enough to require opioid treatment and fo
Original Final Report Due Date: 06/01/2018; Deferral Extension granted per FDA letter dated 01/06/2017
Conduct a pharmacokinetics, safety, and efficacy study of an age-appropriate formulation (oxycodone hydrochloride/acetaminophen) in pediatric patients ages 0 (birth) to less than 2 years with acute pain severe enough to require opioid treatment
Original Final Report Due Date: 07/01/2020; Deferral Extension granted per FDA letter dated 01/06/2017.
Develop an age-appropriate formulation of Qudexy XR (topiramate) extendedrelease capsules that can be used in children ages 1 month to less than 2 years old.
Efforts toward developing an age appropriate formulation are proceeding according to the original schedule.
A study to evaluate the pharmacokinetics (PK) and tolerability of the ageappropriate formulation of Qudexy XR (topiramate) extended-release capsules, developed in PMR 2137-1, as adjunctive therapy in children ages 1 month to less than 2 years w
An adequately controlled study to assess the efficacy and safety of the ageappropriate formulation of Qudexy XR (topiramate) extended-release capsules, developed in PMR 2137-1, as adjunctive therapy in children ages 1 month to less than 2 years
Submit an enhanced pre and post-natal development study (segment III) in rats to demonstrate the safety of dexlansoprazole on developing bone. The studies must include at least 3 dose levels of dexlansoprazole and a control group and should inc
Complete a clinical trial evaluating the potential for Tafinlar (dabrafenib) capsules to prolong the QT/QTc interval in accordance with the principles of the FDA Guidance for Industry entitled “E14 Clinical Evaluation of QT/QTc Interval Prolo
Complete a clinical pharmacokinetic trial to determine the appropriate Tafinlar (dabrafenib) dose in patients with moderate to severe hepatic impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics in Patients w
Good Cause letter sent to sponsor and new milestones ack on 18-NOV-15 due to accrual limitations
Complete a clinical pharmacokinetic trial to determine the appropriate Tafinlar (dabrafenib) dose in patients with severe renal impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics in Patients with Impaired R
Conduct a drug interaction trial to evaluate the effect of rifampin (a strong CYP3A4 and CYP2C8 inducer) on the repeat dose pharmacokinetics of dabrafenib in accordance with the FDA Guidance for Industry entitled “Drug Interaction Studies –
Conduct a clinical trial to evaluate if proton pump inhibitors, H2 antagonists and antacids alter the bioavailability of Tafinlar (dabrafenib) capsules. The worst case scenario can be assessed first to determine if further trials of other gastr
Observational study of incidence and risk factors for uterine perforation among users and particularly when related to breastfeeding and timing of postpartum levonorgestrel intrauterine contraceptive system (IUS) [Mirena] insertion in US women.
Conduct a randomized trial that will characterize the incidence, severity and response to treatment of Tecentriq® induced immune-mediated adverse reactions, including immune-mediated pneumonitis.
Deferred pediatric study of safety and pharmacokinetics (single- and multipledose) under PREA for the relief of moderate to severe pain in patients requiring a continuous, around-the-clock opioid analgesic for an extended period of time in pedi
A randomized, single-blind, multicenter dose-ranging study to obtain pharmacokinetic data and to compare the safety and efficacy of HalfLytely and Bisacodyl Tablet versus NuLYTELY in children (1 - 5 years of age).
Deferred pediatric study under PREA for the treatment of rheumatoid arthritis in pediatric patients ages 2 to 17 years of age.
Study 1 (pJIA), enrollment completed with 52 patients. Study 2 (sJIA), current enrollment 41 patients
Develop an age appropriate formulation of Trokendi XR (topiramate) extendedrelease capsules that can be used in children 1 month to less than 6 years old.
Deferral Extension Requested 03/11/2016.  Denied per FDA letter dated 04/26/2016.
A study to evaluate the pharmacokinetics (PK) and tolerability of an age-appropriate formulation of Trokendi XR (topiramate) extended-release capsules, developed in PMR 2080-1, in children ages 2 years to less than 6 years with partial onset se
The final protocol milestone was missed, because the sponsor has not provided a plan or evidence of formulation development. Deferral Extension Requested November 11, 2015.
A study to evaluate the PK and tolerability of an age-appropriate formulation of Trokendi XR (topiramate) extended-release capsules, developed in PMR 2080-1, as adjunctive therapy in children ages 1 month to less than 2 years with partial onset
An adequately controlled study to assess the efficacy and safety of an age-appropriate formulation of Trokendi XR (topiramate) extended-release capsules, developed in PMR 2080-1, as adjunctive therapy in children ages 1 month to less than 2 yea
The study has not begun but does not meet the criteria for delayed. Deferral Extension Requested November 11, 2015.
Conduct a trial to evaluate pediatric pharmacokinetics, safety and antiviral activity of dolutegravir in HIV-1 infected integrase strand transfer inhibitor-naïve, pediatric subjects 4 weeks to less than 12 years of age. Initial evaluation of d
Per FDA letter dated 06/15/2016, this PMR has been released.
Conduct a trial to evaluate pediatric pharmacokinetics, safety and antiviral activity of dolutegravir in HIV-1 infected subjects, ages 2 years to less than 18 years, who are integrase strand transfer inhibitor (INSTI) experienced with certain I
Deferred study under PREA to evaluate the pharmacokinetics, pharmacodynamics, and safety of pantoprazole in patients 1 month to 11 months of age with erosive esophagitis (EE).
The study/trial has not begun but does not meet the criterion for delayed
Deferred study under PREA to evaluate the pharmacokinetics, healing, maintenance of healing, and symptoms of erosive esophagitis (EE) in patients 1 year to 11 years of age.
Deferred study under PREA to evaluate the pharmacokinetics, healing, maintenance of healing, and symptoms of erosive esophagitis (EE) in patients 12 years to 17 years of age.
Propose and conduct an enhanced pharmacovigilance study of data from clinical trials and all postmarketing sources to assess risk factors for, management of, and consequences of all vascular occlusive events that are serious or require medical
Conduct a pharmacokinetic trial to determine the appropriate dose of Mekinist (trametinib) in patients with hepatic impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics in Patients with Impaired Hepatic Funct
Enrollment complete in the normal and mild hepatic dysfunction groups. Enrollment of moderate and sever groups expected completion June 2017 with trial completion December 2017
A 12-week, randomized, placebo-controlled, dose-ranging efficacy and safety study of mometasone furoate metered dose inhaler (MDI) in the treatment of children ages 5-11 years with persistent asthma.
PMR fulfilled per FDA letter dated 7/12/16.
Conduct a single point vasoconstriction assay (VCA) trial in healthy subjects with adequate bracketing using visual assessment to determine the topical corticosteroid potency classification for Enstilar® (calcipotriene and betamethasone diprop
Deferred pediatric study under PREA to assess the safety and effectiveness of escitalopram oxalate as a treatment of Generalized Anxiety Disorder in pediatric patients ages 7 to 17 (children and adolescents). Both children (ages 7 to 11) and ad
A juvenile rat study to support use of Lexapro in children less than 12 years of age.
A randomized, double-blind, adequately controlled, multiple fixed doses, parallel group clinical study of Dysport (abobotulinumtoxinA) in botulinum toxin-naïve children age 2-17 years with lower extremity spasticity. The minimum duration of th
Conduct a pharmacokinetic trial to determine the appropriate doses of Gilotrif® (afatinib) tablets in patients with moderate and severe renal impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics in Patients
Conduct a prospective, observational study to evaluate the incidence of and risk factors for vascular occlusive events when ponatinib is given with or without anticoagulant or antiplatelet agents. Submit a protocol that includes measures to ens
Provide long-term follow-up of all patients enrolled in the Phase 1 (AP24534-07-101) and Phase 2 (AP24534-10-201) clinical trials. Assess the long-term safety of ponatinib treatment, including the long-term risk of vascular occlusive events. In
Prepare and submit an integrated safety data and summary (final report submission) from all three clinical trials cited in PMRs 2113-3 and 2113-4 (Phase 1, Phase 2, and Phase 3). Include narratives for all cases of vascular occlusion.
Conduct a randomized, multi-arm trial to characterize the safety of a range of ponatinib doses. The trial should be of sufficient size and duration to inform safe use of Iclusig in chronic phase CML. The trial should also assess the efficacy of
To determine the potential for pharmacokinetic interactions between Erbitux (cetuximab) and carboplatin you will conduct the drug-drug interaction clinical trial, Study I4E-MC-JXBB.
Deferred pediatric study under PREA to determine the safety of Zemplar (paricalcitol) for the treatment of secondary hyperparathyroidism associated with chronic kidney disease (CKD) Stage 5 in pediatric patients ages 0 to 9 years receiving peri
Conduct a prospective, observational study of 250 pregnant women with asthma exposed to Omalizumab that will assess the outcomes in the offspring born to those women who were exposed to Omalizumab during pregnancy and breastfeeding relative to
The study completion and final study report milestones were missed. 309 patients have been enrolled.
Assessment of pharmacokinetic (PK/PD) parameters and dosing, safety, tolerance and immunogenicity in the pediatric population 2 years to < 17 years with polyarticular JIA.
Final report submitted to FDA on 5/27/16
A randomized, double-blind, placebo-controlled trial evaluating the effect of Trulicity (dulaglutide) on the incidence of major adverse cardiovascular events (MACE) in patients with type 2 diabetes mellitus. The primary objective of the trial s
Conduct an 18-month study in dogs to evaluate the impact of cytoplasmic vacuoles in the adrenal gland and the aortic outflow tract of the heart.
A juvenile rat toxicology study is required to identify the unexpected serious risk of adverse effects of Botox (onabotulinum toxin type A) on postnatal growth and development.  The study should utilize animals of an age range and stage(s) of d
The Sponsor is currently negotiating a revised study protocol with the FDA.
Expand the Pediatric IBD Registry (DEVELOP) to include pediatric patients with ulcerative colitis (UC) and indeterminate colitis (IC).
Conduct a multi-center, randomized, blinded, vehicle-controlled study, including pharmacokinetic assessments, with luliconazole cream 1% for the treatment of tinea corporis in pediatric patients 2 years of age and older.
MEDICIS PHARMACEUTICALS A DIV OF VALEANT PHARMACEUTICALS NORTH AMERICA LLC
Conduct a maximum use pharmacokinetic safety study in pediatric patients 12 years to 17 years 11 months of age with interdigital tinea pedis and tinea cruris.
Conduct an in vivo drug interaction trial using an appropriate probe substrate to evaluate the inhibition potential of luliconazole for CYP2C19 under maximal use conditions in subjects with tinea cruris and interdigital tinea pedis.
Conduct an in vivo drug interaction trial using an appropriate probe substrate to evaluate the inhibition potential of luliconazole for CYP3A4 under maximal use conditions in subjects with tinea cruris and interdigital tinea pedis. This trial m
Conduct in vitro assessments to evaluate the following: a. Inhibition potential of luliconazole for enzymes CYP2B6 and CYP2C8 b. Induction potential of luliconazole for enzymes CYP1A2, CYP2B6 and CYP3A Further in vivo assessment to address drug
Complete and submit the final results of the ongoing randomized, double-blind, placebocontrolled Phase 3 clinical trial (PCI-32765MCL3002) of ibrutinib in combination with bendamustine and rituximab in patients with newly diagnosed mantle cell
Determine the effect of a broad range of concentrations of ibrutinib on the potential to inhibit platelet function by conducting in vitro studies. Assessment methods should include evaluation of effects on platelet aggregation, including GPIb-m
Conduct an assessment and an analysis of data from clinical trials and all post-marketing sources in order to characterize the risk of serious bleeding in patients treated with Imbruvica®, (ibrutinib) Capsules. The risks of special interest ar
Determine the effect of ibrutinib on the QT/QTc interval in healthy subjects on one or more therapeutic dose levels. Conduct and submit results of a thorough QT trial to evaluate the effects of ibrutinib on the QT /QTc interval.
An observational study (N = 1200) to assess the long-term safety of radium Ra 223 dichloride 50 kBq/kg every 4 weeks for 6 doses in patients with castrationresistant prostate cancer with bone metastases.
A randomized clinical trial to assess the safety of radium Ra 223 dichloride in patients with castration-resistant prostate cancer, symptomatic bone metastases and no known visceral metastatic disease.
A trial of the short and long-term safety of re-treatment of patients with castration-resistant prostate cancer with bone metastases with radium Ra 223 dichloride.
Optimize the dosing regimen of Xofigo by conducting a randomized Phase 2 clinical trial to evaluate the efficacy and safety of Xofigo at a dose higher than 50 kBq/kg in patients with castration-resistant prostate cancer with bone metastases. De
Evaluate for cross-reactivity of anti-ecallantide antibodies with TFPI, perform studies to determine if human anti-ecallantide antibodies bind TFPI, and perform suitability studies and epitope mapping of the human anti-ecallantide antibody resp
Conduct a study in pediatric patients, "A Phase II Open-Label Multi-Center Study to Assess the Safety and Efficacy of Certolizumab pegol in Children and Adolescents with Active Crohn's Disease" [Study CDP870-035]. This study is proposed to eval
Original final report due date: 10/31/13. Deferral extension granted until 6/30/19 per FDA letter dated 3/18/14.
A long-term observational study in the U.S. that will include approximately 2000 CIMZIA-treated Crohn's disease patients and 2000 matched controls receiving other treatments for Crohn's disease. Patients will be monitored for ten years.
Conduct pediatric PK, dose selection, safety, and treatment trial(s) in patients from 2 to < 18 years of age. Initial evaluation of telbivudine exposure must be performed in either a pilot trial or lead-in subtrial to allow dose selection, foll
Conduct pediatric PK, dose selection, and treatment trial(s) in patients from birth to < 2 years of age. Trials in pediatric patients 2 to < 18 years age should be completed before determining whether it is appropriate to study telbivudine for
Submit the results of overall survival, along with datasets and analysis programs, after 240 OS events have occurred from Trial 14295, “A Double-Blind, Randomized Phase III Study Evaluating the Efficacy and Safety of Sorafenib Compared to Pla
Complete a pharmacokinetic trial to determine the appropriate dose of Zykadia (ceritinib) in patients with hepatic impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics in Patients with Impaired Hepatic Functi
Sponsor notified Agency of likely delay with FSR due to inadequate subjects accrued in Jan 16– more subjects needed in severe hepatic group. Failure to Show Good Cause letter sent to sponsor 3-OCT-16.
Conduct a clinical trial to evaluate the effect of repeat doses of Zykadia (ceritinib) on the single dose pharmacokinetics of midazolam (a sensitive CYP3A4 substrate) in accordance with the FDA Guidance for Industry entitled “Drug Interaction
Conduct a clinical trial to evaluate the effect of repeat doses of Zykadia (ceritinib) on the single dose pharmacokinetics of warfarin (a sensitive CYP2C9 substrate) in accordance with the FDA Guidance for Industry entitled “Drug Interaction
Conduct a clinical trial to evaluate if proton pump inhibitors, H2-receptor antagonists, and antacids alter the bioavailability of Zykadia (ceritinib) and to determine how to dose Zykadia (ceritinib) with regard to concomitant gastric acid redu
Conduct a single dose pharmacokinetic (PK) study in children from 3 months to less than 12 years of age.
Final report submitted on March 31, 2016.
Conduct a single dose PK study in neonates/infants from 0 to less than 3 months of age.
Conduct a Phase 3, randomized, comparator-controlled study of dalbavancin in children from 3 months to 17 years of age with ABSSSI.
Study completion date is December, 2016.
Conduct a Phase 3, randomized, comparator-controlled study of dalbavancin in neonates/infants from birth to less than 3 months of age with ABSSSI.
Conduct a trial in patients, ages 2 to < 18 years, to evaluate the pharmacokinetic (PK), safety, and tolerability of two new formulations of posaconazole (IV solution and/or new  age-appropriate oral formulation) in immunocompromised pediatric
Conduct a comparative, double-blind, randomized, multi-center trial, in patients ages 2 to < 18 years, to evaluate the safety, efficacy, and tolerability of posaconazole for the prophylaxis of invasive fungal infections (IFI) in pediatric patie
Final protocol submission date is September, 2017.
Submit integrated analyses for genotypic and phenotypic resistance for studies AI463048, AI463050, AI463085, AI463901, AI463110, and AI463111, and integrated phenotypic resistance analyses for studies AI463028 and AI463189 in SAS format. The vi
A prospective, observational, controlled, pregnancy exposure registry study to monitor pregnancies exposed to apremilast with the primary objective to evaluate whether there is any increase in the risk of birth defects.
Pediatric studies under PREA for the treatment of pediatric patients aged two years and older with detrusor overactivity associated with a known neurological condition (e.g., spina bifida).
Original Final Report Due Date: 03/31/2014; Deferral Extension granted per FDA letter dated 03/24/2014
A study to evaluate the safety of metronidazole gel 1.3% single dose in the treatment of bacterial vaginosis in females 12-<18 years of age.
Conduct a prospective, observational registry study of women with Crohn's disease, rheumatoid arthritis and psoriatic arthritis exposed to Infliximab during pregnancy.  This study will assess the pregnancy outcomes in women who were exposed to
Assess the safety and efficacy of Peginterferon alfa-2a versus a no-treatment control in 110 pediatric HBeAg positive patients chronically infected with the hepatitis B virus, who have compensated liver disease.
Original Final Report Due Date: 11/30/2013; Deferral Extension granted per FDA letter dated 04/04/2016.
An assessment and analysis of spontaneous reports of inadvertent exposure of anyone other than the patient who has been exposed to Valchlor (mechlorethamine) gel 0.016%. Specialized follow-up should be obtained on these cases to collect additio
A long-term prospective observational study (product exposure registry) to evaluate potential serious hepatic risks related to the use of Opsumit (macitentan). The registry will include a sample of patients prescribed Opsumit (macitentan) and e
An assessment and analysis of reports of serious hepatic adverse events (to be defined in the protocol) in patients treated with Opsumit (macitentan) submitted according to the schedule outlined below. Specialized follow up should be obtained o
Submit the final report and datasets for the ongoing trial GS-US-334-0154, entitled, “A Phase 2b, Open-Label Study of 200 mg or 400 mg Sofosbuvir + RBV for 24 Weeks in Genotype 1 or 3 HCV-Infected Subjects with Renal Insufficiency”, in orde
Submit the final report and datasets for the ongoing trial GS-US-334-0109, entitled, “An Open-Label Study of GS-7977 + Ribavirin with or without Peginterferon Alfa-2a in Subjects with Chronic HCV Infection Who Participated in Prior Gilead HCV
Conduct a trial to quantify efavirenz QT prolongation in CYP2B6*6 homozygous and heterozygous subjects.
Develop and validate an appropriate analytical method for determining the individual component phytosterol content in Liposyn III 20% IV Fat Emulsion.
The final report due date has passed with no final report received
Liposyn® III (Soybean Oil [Fat Emulsion])
Test for the individual component phytosterol content in all batches of Liposyn III 20% IV Fat Emulsion, manufactured over a three year period, using the method developed under PMR 2143-1. Based on these test results, establish limits for each
Develop and validate an appropriate analytical method for measuring phytosterol levels in plasma.
Randomized controlled trial in pediatric patients, including neonates, comparing Liposyn III 20% IV Fat Emulsion with a phytosterol-depleted formulation of Liposyn III 20% IV Fat Emulsion to evaluate the incidence of liver injury, including eit
Provide data analyses from the Nordic Database Initiative regarding the occurrence of serious infection, tuberculosis, opportunistic infections, malignancy, hypersensitivity reactions, autoimmune disease, neurologic or demyelinating disease, ca
Establish a U.S.-based prospective, observational pregnancy exposure registry that compares the pregnancy and fetal outcomes of women exposed to Stelara¿ (ustekinumab) during pregnancy to an unexposed control population.  Outcomes of the regis
The original Final Report submission milestone (7/15/2014) was missed. (As of 6/30/2016: 13 patients exposed)
A randomized and controlled pediatric study under PREA to evaluate the safety, efficacy, and pharmacokinetics of Tanzeum (albiglutide) for the treatment of type 2 diabetes mellitus in pediatric patients ages 10-17 years (inclusive).
The final protocol submission milestone was missed, because protocol negotiations were ongoing.
An assessment and analysis of spontaneous reports of serious risks related to the use of Myalept (metreleptin) including: fatal or necrotizing pancreatitis, hepatic adverse events, severe hypoglycemia, serious hypersensitivity reactions, seriou
Conduct a trial in patients, ages 2 to < 18 years, to evaluate the pharmacokinetic (PK), safety, and tolerability of two new formulations of posaconazole (IV solution and/or new ageappropriate oral formulation) in mmunocompromised pediatric pat
Complete a randomized, multicenter, parallel, single-dose study to explore the pharmacokinetics (PK) and pharmacodynamics (PD) of dapagliflozin in children, 10 to 17 years of age with type 2 diabetes mellitus (T2DM) receiving one of the three d
Per FDA letter dated 03/21/2016, this PMR has been fulfilled.
A 26-week randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of dapagliflozin for the treatment of pediatric subjects ages 10 to <18 years of age with type 2 diabetes mellitus (T2DM), as add-on
Deferral Extension Requested 02/08/2016.  Denied per FDA letter dated 03/18/2016.
Conduct a study to evaluate dapagliflozin in an orthotopic rodent bladder tumor promotion model.
The final protocol submission milestone was missed because an additional exploratory study was needed prior to finalizing the protocol for the definitive study. FDA determined that the applicant demonstrated “good cause” for the delay.
An assessment and analysis of all foreign and domestic spontaneous reports of serious hepatic abnormalities and pregnancy outcomes in patients treated with dapagliflozin. The enhanced pharmacovigilance study should continue for 5 years.
A randomized, double-blind, placebo-controlled trial (the DECLARE trial) evaluating the effect of dapagliflozin on the incidence of major adverse cardiovascular events (MACE) in patients with type 2 diabetes mellitus. The primary objective of t
To assess the risk of bladder cancer associated with dapagliflozin, conduct adequate follow-up beyond completion of the cardiovascular outcomes trial (DECLARE) to observe a total of 66 events of bladder cancer, with 80% power to exclude a relat
The final protocol submission milestone was missed because the applicant proposed to delay protocol finalization until more of the primary cardiovascular events have been reached in the cardiovascular outcomes trial to allow for a more robust a
Conduct a study to evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response) of Olysio™ (simeprevir) as a component of a combination antiviral treatment regimen in pediatric subjects 3 through 17 years
Long-term safety follow-up in patients with myelofibrosis and lower baseline thrombocytopenia who are receiving longer term treatment with Jakafi. This longer term safety analysis may be based on further followup of ongoing trials or you may pr
Conduct in-vitro assessments to evaluate the following: 1. Inhibition potential of econazole nitrate for enzymes CYP1A2, 2B6, 2C8, 2C9, 2C19, 2D6 and 3A4. 2. Induction potential of econazole nitrate for enzymes CYP1A2, 2B6 and 3A. Further in-vi
A juvenile dog toxicology study under PREA to identify and characterize the unexpected serious risk of adverse effects of eslicarbazepine acetate on the immune system of the developing organism. The study should utilize animals of an age range
Per FDA letter dated 03/22/2016, this PMR has been fulfilled.
A pharmacokinetic and tolerability study in pediatric patients ages 1 month to < 24 months with partial-onset seizures. At least two maintenance dose levels of eslicarbazepine acetate must be evaluated to characterize pharmacokinetic parameters
A prospective, randomized, controlled, double-blind, efficacy and safety study of eslicarbazepine acetate in children ages 12 years to <18 years for the adjunctive the treatment of partial onset seizures. The primary efficacy endpoint must exam
The final protocol milestone was missed. Draft protocols under FDA review.
Evaluate the long-term safety of eculizumab by enrollng all patients with aHUS treated with eculizumab in a Soliris registry program and analyzing outcomes for a period of no less than five years. Submit interim reports annually to the BLA incl
Deferred pediatric study under PREA for the treatment of major depressive disorder in pediatric patients aged 7 to 17. Conduct a study to obtain pharmacokinetic, safety, and tolerability data and provide information pertinent to dosing vortioxe
Due to delay in enrollment, agreement was obtained to submit the final report by April 2015. Final study report submitted on 29 Apr 2015.
Deferred pediatric study under PREA for the treatment of major depressive disorder in children aged 7 to 11years. Conduct a study to obtain data on the efficacy and safety of vortioxetine in the relevant pediatric population. This must be a pla
This study has not been initiated, but does not meet the criterion for delayed.
Deferred pediatric study under PREA for the treatment of major depressive disorder in adolescents aged 12 to17 years. Conduct a study to obtain data on the efficacy and safety of vortioxetine in the relevant pediatric population. This must be a
In-vivo pharmacokinetic trial in subjects with severe hepatic impairment compared to healthy subjects using the 5 mg dose.
A controlled trial to evaluate the longer-term (i.e., maintenance) efficacy of vortioxetine in the treatment of adults with major depressive disorder in the US. This trial must include a placebo group and several fixed doses and must utilize a
Collect long-term safety data for subjects enrolled in the pediatric simeprevir safety, pharmacokinetics and efficacy study. Data collected should include at least 3 years of follow-up in order to characterize the long-term safety of Olysio™
Submit the final report and datasets from the ongoing clinical trial TMC435HPC3005 entitled “A Phase 3, Randomized, Double-Blind, Double Dummy, Placebo-Controlled Study Conducted in the Asia-Pacific Region to Investigate the Efficacy, Pharmac
Deferred pediatric study under PREA for the maintenance treatment of Major Depressive Disorder (MDD) in subjects age 7 to 17 years
To conduct a prospective, observational study in at least 5000 subjects with multiple sclerosis who are receiving Natalizumab, with each subject followed for at least 5 years, by completing protocol 101-MS-402, "TYGRIS:  TYSABRI® Global Observ
Delayed due to study completion date and final report submission date.
Conduct a single dose pharmacokinetic (PK) trial in patients =/>1 to 17 years old.
Deferral Extension Requested 05/25/2016.  Denied per FDA letter dated 06/29/2016.
Conduct a single dose PK trial in neonates/infants 0 to <1 year old.
Final protocol submission date is September, 2016
Conduct a Phase 3, randomized, comparator-controlled trial of telavancin in children from birth to 17 years old with gram positive infections.
Final protocol submission date is 12/2015
Deferred pediatric study under PREA: Conduct a pharmacokinetic and safety study of an age-appropriate formulation of hydrocodone extended-release in patients from ages 12 to less than 17 years with moderate-to-severe pain requiring around the c
Deferred pediatric study under PREA: Conduct a pharmacokinetic and safety study of an age-appropriate formulation of hydrocodone extended-release in patients from ages 7 to less than 12 years with moderate-to-severe pain requiring around the cl
Conduct one or more studies to provide quantitative estimates of the serious risks of misuse, abuse, addiction, overdose, and death associated with long-term use of opioid analgesics for management of chronic pain, among patients prescribed ER/
Develop and validate measures of the following opioid-related adverse events: misuse, abuse, addiction, overdose and death (based on DHHS definition, or any agreed-upon definition), which will be used to inform the design and analysis for PMR #
Conduct a study to validate coded medical terminologies (e.g., ICD9, ICD10, SNOMED) used to identify the following opioid-related adverse events:misuse, abuse, addiction, overdose, and death in any existing post-marketing databases to be employ
Conduct a study to define and validate “doctor/pharmacy shopping” as outcomes suggestive of misuse, abuse and/or addiction. These validated codes will be used to inform the design and analysis for PMR # 2065-1.
Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following use of ER/LA opioid analgesics for at least one year to treat chronic pain. We strongly encourage you to use the same trial to assess the develo
Conduct US surveillance studies for five years from the date of marketing DALVANCE to determine if resistance to dalbavancin has developed in those organisms specific to the indication in the label for ABSSSI.
Conduct studies to define the mechanism(s) of resistance for isolates identified as being resistant to dalbavancin during the surveillance period (five years from the date of marketing).
Deferred pediatric study under PREA to evaluate the pharmacokinetics, pharmacodynamics, and safety of esomeprazole strontium for healing and maintenance of healing of erosive esophagitis (EE) in patients 1 month to 17 years, inclusive. The stud
Deferred pediatric study under PREA to evaluate the safety of esomeprazolen strontium for treating symptomatic gastroesophageal reflux disease (GERD) in patients 1 year to 17 years, inclusive. The study must include an adequate number of patien
Deferred pediatric study under PREA to evaluate the pharmacokinetics, pharmacodynamics, and safety of esomeprazole strontium for reducing the risk of NSAIDassociated gastric ulcer in patients 2 years to 17 years, inclusive. The study must inclu
Deferred pediatric study under PREA to evaluate the safety and efficacy of esomeprazole strontium in combination with clarithromycin and amoxicillin for the eradication of Helicobacter pylori in symptomatic pediatric patients 2 to 17 years, inc
In vitro studies to evaluate the potential inhibitory effects of Kuvan (sapropterin dihydrochloride) activity of major cytochrome P450 enzymes including, but not limited to, CYP3A4 to assess drug interaction potential in humans
In vitro studies to evaluate if Kuvan (sapropterin dihydrochloride) is an inducer of CYP3A4 to assess drug interaction potential in humans
In vitro studies to evaluate the potential effects of Kuvan (sapropterin dihydrochloride) on activity of transporters including p-glycoprotein and breastcancer- resistance protein to assess drug interaction potential in humans
Submit data from controlled extraction studies of [...] to qualitatively and quantitatively determine the chemical species which may migrate into the drug product solution.
Submit leachable data from long-term stability studies with the final drug product formulation (taking into consideration the proposed shelf-life) to qualitatively and quantitatively determine the chemical species which migrate into the drug pr
A multi-center observational study of Humira (adalimumab) in adults with moderately to severely active ulcerative colitis treated in a routine clinical setting, to assess the long-term safety as measured by the incidence of opportunistic infect
A randomized, double-blind, adequately controlled, multiple fixed doses, parallel group clinical study of Dysport (abobotulinumtoxinA) in botulinum toxin-naïve adults with lower extremity spasticity. The minimum duration of the study is 12 wee
Conduct a cell culture study to characterize the antiviral activity of ombitasvir against representative HCV subtype 4b isolates, including those with amino acid variability (relative to subtypes 4a and 4d) at NS5A positions 30 and 93.
Conduct a nationally representative drug utilization study of sufficient detail to characterize use of OxyContin in children aged 17 years and younger. The data from this study will provide a denominator for the risks assessed in PMR #2923-1 an
Evaluate the long-term safety of Vimizim in adult and pediatric patients enrolled in the Morquio A Registry for a period of ten years, including but not limited to the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes i
A study to reanalyze banked immunogenicity serum samples from ulcerative colitis trial C13006 and Crohn’s disease trial C13007 to determine the presence of anti-drug antibodies (ADA) using an improved ADA assay format with reduced sensitivity
To assess the safety of long-term use of rilonacept in the pediatric patient population by establishing a pediatric registry.  The registry will collect information on growth and development as well as adverse events, particularly serious infec
To commit to providing complete validation data for the anti-Oncaspar ELISA assay. The validation studies will provide an assessment of the sensitivity (in mass units of antibodies), specificity, and reproducibility of the assay. The cutpoint f
To develop validated screening and confirmatory assays to assess for the presence of anti-tbo-filgrastim antibodies.  The validation of the assay should include the sensitivity and specificity for detection of anti-tbo-filgrastim antibodies tha
To conduct an assessment for the presence of anti- tbo-filgrastim and anti-native human G-CSF binding antibodies using the validated assays developed under PMR 2 in at least 426 patients enrolled/to be enrolled in one or more clinical trials, a
Centocor commits to designing and implementing a registry of patients with pediatric Crohn's disease being treated with REMICADE that will be established to obtain long-term clinical status and safety information.  Information will be collected
Submit revised labeling, which accurately reflects the immune response to  Sargramostim.
To conduct clinical trial 20070782 entitled "A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Long-term. Safety and Efficacy of Darbepoetin Alfa Administered at 500 ug Once-Every-3-Weeks (Q3W) in Anemic subjects with Advance
The Sponsor had difficulties with patient enrollment.The patient accrual milestone has been missed.The study is delayed. Original milestones: patient accrual by July 2016; trial completion June 30, 2019; Final report and database submission Dec
To conduct a verification trial to describe clinical benefit attributable to Elaprase (idursulfase) in a cohort of Hunter syndrome patients 5 years of age and younger.At a minimum, this trial will assess longitudinal changes in anthropometric m
To conduct a clinical trial of Herceptin (trastuzumab) in combination with standard chemotherapy, in patients with previously untreated, HER2 overexpressing, metastatic gastric cancer, if the pharmacokinetic trial (PMR #2) definitively demonstr
Shire commits to evaluating long-term safety and efficacy data in an observational survey (the Hunter Outcome Survey, HOS) of patients with Hunter syndrome being treated with ELAPRASE.  In addition to clinical and laboratory tests that are part
Pregnancy registry to evaluate pregnancy outcomes for women exposed to Actemra (tocilizumab) during pregnancy.  Utilize the established Organization of Teratology Information Specialists (OTIS) pregnancy registry to evaluate pregnancy outcomes.
To evaluate a prophylactic immune tolerance regimen in a cohort of Hunter syndrome patients treated with Elaprase (idursulfase) who are at high risk of developing persistent neutralizing antibody that could result in diminished clinical benefit
Conduct Study Protocol P10-262, an 800-patient observational study, with inclusion of a reference group, of pediatric patients 4 to 17 years of age with moderately to severely active polyarticular juvenile idiopathic arthritis (JIA).
Enrollment completed on 1/2014 (846 patients). 12/29/15 Final Study Report due date revised to be submitted on 12/2024.
Review post-marketing adverse event surveillance data after one year of commercial distribution and propose revised labeling as warranted.
Evaluate the pediatric pharmacokinetics (PK), safety, and antiviral activity of once daily elvitegravir combined with a background regimen including a protease inhibitor coadministered with ritonavir in HIV-1 treatment-experienced pediatric sub
No longer enrolling for the study- See section D for further explanation.
Randomized, double-blind, adequate and well controlled, multiple fixed-dose, parallel group clinical trial of Xeomin (incobotulinumtoxinA) in botulinum toxin-naive children age 2-17 years with lower extremity spasticity.  The minimum duration o
Merz Pharmaceuticals GmbH c/o Merz Pharmaceuticals LLC
Enhanced pharmacovigilance program for reports of malignancy in pediatric, adolescent, and young adult (¿ 30 years of age) patients treated with Remicade (infliximab), for a period of up to 10 years after this notification to collect data that
A randomized, controlled trial evaluating the efficacy, safety, and pharmacokinetics of sugammadex injection when used to reverse neuromuscular blockade induced by either rocuronium or vecuronium must be conducted in pediatric patients ages bir
Conduct a postmarketing study to analyze the demographic characteristics, concomitant medication use, and comorbid conditions in patients who did not respond to sugammadex reversal in the development program, in postmarket studies that have bee
Conduct a postmarketing clinical trial comparing sugammadex to placebo and/or drugs approved for the management of the reversal of the effects of neuromuscular blockade induced by rocuronium or vecuronium in a population of American Society of
Conduct a postmarketing clinical trial comparing sugammadex to placebo and/or drugs approved for the management of the reversal of the effects of neuromuscular blockade induced by rocuronium or vecuronium in patients with morbid obesity. The go
Continue the registry of patients with Fabry disease being treated with Agalsidase beta that was established to obtain long-term clinical status information.  This study will be revised so that detailed clinical status information is collected
A long-term follow-up study to evaluate long-term efficacy and immunogenicity outcomes in a subpopulation of patients in LOTS and LOTS Extension Studies whose response to Lumizyme (alglucosidase alfa) is associated with substantial improvement
As part of the ongoing Pompe Registry, prospective outcome data will be collected in patients enrolled in the Registry to assess the long-term efficacy of Lumizyme (alglucosidase alfa).
Conduct a dose-ranging study to determine the pharmacokinetics/ pharmacodynamics, safety, and tolerability of Entyvio (vedolizumab) in pediatric patients 5 to 17 years of age with moderately to severely active ulcerative colitis or Crohn’s di
To conduct a study to determine the incidence of cataract surgery associated with Palifermin administration. This study will be a component of the Phase 4 prospective cohort study using the available Center for International Blood and Marrow Tr
A prospective safety study conducted within the ongoing Pompe Registry to assess the known serious risks of anaphylaxis and severe allergic reactions, and signals of severe cutaneous and systemic immune complex-mediated reactions with Lumizyme
CONFIRMATORY TRIAL - A randomized phase 3 trial of SGN-35 (brentuximab vedotin) in combination with AVD versus ABVD as frontline therapy in patients with advanced Hodgkin Lymphoma. Enrollment of at least 880 patients is expected with a primary
Conduct a study to determine the incidence of cataracts and decreased visual acuity associated with Palifermin administration.  This study will be a component of the clinical study 20040253 conducted in patients with metastatic breast cancer re
Conduct a prospective cohort study using the available International Bone Marrow Transplant Registry (IBMTR) and Autologous Blood and Bone Marrow Registry (ABMTR) databases to evaluate the incidence of secondary malignancies, cancer relapse rat
Submit the final efficacy (disease-free survival) and safety results from Trial BO25126 (APHINITY) as defined in your protocol and Statistical Analysis Plan (SAP).
A long-term observational study in administrative databases to prospectively evaluate the incidence of serious infection including skin infection, dermatologic adverse events, and over-suppression of bone turnover in postmenopausal women admini
Randomized, double-blind, adequately controlled, multiple fixed dose, parallel group clinical trial of Botox (onabotulinum toxin type A) in botulinum toxin-naive children age 2- 17 years with upper extremity spasticity.  The minimum duration of
The final report milestone was missed, because the FDA was engaged in discussion with the applicant regarding the study design at the time the final report submission milestone was due and FDA determined that the applicant demonstrated good cau
Provide information on maintenance of response with dosing intervals longer than every 12 weeks among relevant populations (e.g., subjects whose psoriasis is cleared as measured by PGA and PASI or who have minimal psoriasis).  This information
Submit safety data assessing distant spread of toxin effects after multiple administrations of Xeomin (incobotulinumtoxinA), during a minimum period of 12 months, collected in at least 100 adult patients. Approximately one half of the patients
Conduct a Phase 4 study to evaluate the effect of raxibacumab on immunogenicity of anthrax vaccine.
Conduct a pregnancy registry to evaluate pregnancy outcomes for women exposed to Benlysta (belimumab) during pregnancy.
Submit a final report for the long-term, open-label, continuation trial LBSL99.
Submit a final report for the long-term, open-label, continuation trial C1074.
Recruitment ongoing, 738 subjects enrolled. Revised milestone letter issued 10/21/14. Final report submission October 2016.
To conduct a Phase 1 study, Protocol 20050252 entitled, "A Phase 1 Study to Evaluate the Safety and Pharmacokinetics of Panitumumab in Children with Refractory Solid Tumors" in children and adolescents (up to 18 yr of age) to provide the initia
A prospective, long-term observational study that will collect safety data, including sexual dysfunction and immunogenicity, from subjects previously treated with XIAFLEX® (collagenase clostridium histolyticum) who were enrolled and treated in
Enhanced pharmacovigilance program for reports of malignancy in pediatric, adolescent, and young adult (¿  30 years of age) patients treated with Cimzia (certolizumab pegol), for a period of up to 10 years after this notification to collect da
A study to bank samples for future evaluation to identify genetic mutations and other biomarkers that predispose inflammatory bowel disease (IBD) patients to developing Hepatosplenic T-Cell Lymphoma (HSTCL).
To conduct study protocol P06-134, a 5-year, 5000 patient, multi-center, uncontrolled, observational study of adult patients with Crohn's disease treated in a routine clinical setting with adalimumab.
Perform, complete and submit the full study report for a single-dose study of pharmacokinetics and pharmacodynamics (PK/PD) of edoxaban in pediatric patients at risk for venous thromboembolism (VTE), requiring anticoagulation or recently comple
First subject dosed on Nov 4, 2014; 8 subjects enrolled as of Jan 7, 2016..
Perform, complete and submit the full study report for a phase 3 multicenter, randomized, active control study of edoxaban in pediatric patients with documented venous thromboembolism in accordance with your October 31, 2013 agreed upon Initial
Final Protocol Submission is due 12/14/2016.
A juvenile rat toxicology study is required to identify the unexpected, serious risk of adverse effects of Xeomin (incobotulinumtoxinA) on postnatal growth and development.  The study should utilize animals of an age range and stage(s) of devel
Phase 2, multicenter study to evaluate the safety, efficacy, and pharmacokinetics of belimumab plus background standard therapy in 100 pediatric subjects ages 5 years to 17 years of age with active systemic lupus erythematosus (SLE).
Per FDA letter dated 11/14/2016, this PMR has been released..
To conduct a trial that will evaluate the safety, efficacy, PK/PD and immunogenicity of IV golimumab in pediatric patients between the ages 2 to 17 years and 11 monthswith active juvenile idiopathic arthritis (JIA) despite standard therapy with
The final protocol milestone was missed because data analysis was not complete.
To conduct a phase 4, randomized, controlled, open label, multicenter parallel group study to assess the all cause mortality and cardiovascular morbidity in anemic patients with chronic kidney disease who are on dialysis and not undergoing dial
2,402 patients have been enrolled in the study. Enrollment was complete Nov 8, 2011. The final report was due September 29, 2012. Extended to January 2021.
Mircera® (Methoxy Polyethylene Glycol-Epoetin Beta)
CONFIRMATORY TRIAL- A randomized phase 3, double-blind, placebo-controlled trial of SGN-35 (brentuximab vedotin) in combination with CH-P versus CHOP as frontline therapy in patients with CD30-positive mature T- and NK-cell lymphomas including
Unless FDA agrees, based on your completion of Postmarketing Commitment Number 1, that it is not appropriate to conduct a pediatric study in pediatric patients age 6 to 11 years, after completing the study described in Postmarketing Commitment
Deferral Extension Granted per FDA letter dated 07/16/2013.
Conduct a prospective, observational registry study of women with rheumatoid arthritis, juvenile rheumatoid arthritis, psoriatic arthritis and plaque psoriasis exposed to Etanercept during pregnancy or within two weeks prior to conception.  Thi
A prospective pharmacokinetic (PK) study will be conducted to characterize the pharmacokinetics of Lumizyme (alglucosidase alfa) in pediatric patients in the age range of 8 years to 18 years.
The final report submission milestone of 3/30/2015 was missed. The sponsor is working with the FDA to discuss options to modify the protocol. Two patients were enrolled in the study as of the end of the reporting period.
Genzyme commits to designing and implementing an immune tolerance protocol in Pompe disease patients who have significant antibody titers, or the presence of neutralizing antibody, and are failing treatment.  Genzyme commits to designing and im
FDA issued a not fulfilled letter 6/20/2014. The sponsor is working with the FDA to discuss options to fulfill the PMC.
A long-term surveillance study in postmenopausal women administered Prolia (denosumab) to prospectively evaluate the incidence of serious infection including skin infections, dermatologic adverse events, and over-suppression of bone turnover.
Evaluate the pharmacokinetics, safety and antiviral activity (efficacy) of abacavir/dolutegravir/lamivudine FDC tablets in HIV infected pediatric subjects 12 years to less than 18 years of age and weighing at least 40 kg. The safety and antivir
BioMarin is required to conduct the following assessments using data from patients enrolled in the ongoing MPS I Registry.  Immunogenicity will be studied using validated anti-Aldurazyme IgG and IgE, and neutralizing assays.  The relationship b
BioMarin commits to developing an assay to detect anti-Aldurazyme antibody mediated neutralization of enzyme uptake. Such an assay could be developed based on the current cell uptake assay already used for Aldurazyme lot release. BioMarin commi
To assess the serious risks of respiratory depression, accidental injury, overdose, misuse, accidental exposure, and medication errors associated with the use of OxyContin in opioid-tolerant pediatric patients aged 11-17, and to assess the seri
To develop, validate, and implement a ligand binding assay to supplement the neutralizing bioassay that tests for the presence of neutralizing antibodies in serum samples from patients with generalized lipodystrophy.
To conduct a randomized, multicenter, observation-controlled trial of peginterferon alfa-2b as adjuvant therapy in 1200 patients with ulcerated primary cutaneous melanoma with negative sentinel lymph node biopsy (T1b-T4bN0M0) randomized 1:1 to
Submit a report of the immunogenicity evaluation of all subjects enrolled in studies of Sargramostim.
To develop a validated cross-reactive immunologic material (CRIM) assay for patients with Hunter syndrome and test patient samples in a cohort of patientsprior to Elaprase (idursulfase) treatment. Results will be correlated with antibody respon
The study completion milestone was missed. The sponsor has been working on a revised protocol with the FDA.
To conduct a multi-center, randomized, controlled, parallel-group study to confirm the optimal methoxy polyethylene glycol-epoetin beta dosage when used for the maintenance treatment of anemia in pediatric patients ages 5 to 17 years who have c
Final report is deferred to August 2018.
Conduct a one-year, randomized, placebo-controlled clinical trial with Benlysta (belimumab) in 5,000 patients with active, autoantibody-positive systemic lupus erythematosus to evaluate Benlysta's long term safety profile including adverse even
A JIA patient safety registry comprised of at least 500 patients.  The protocol for this study should include a plan for more intensive scrutiny for the first 3 years, with annual follow ups (which could be telephonic) assessing for occurrence
Missed final protocol submission date 12/2008. Delay in study start. As of 12/22/2015, enrollment 295, target 500. First interim report submitted on time in 6/2014.
A safety and pharmacokinetic trial as a substudy of the DEVELOP registry to evaluate whether trough concentrations at the time of loss of clinical response can be used to identify pediatric UC and Crohn¿s disease patients who have low inflixim
Assess the pharmacokinetics, safety, immunogenicity, and efficacy of golimumab in pediatric patients 2 to 16 years of age with active polyarticular juvenile idiopathic arthritis (pJIA).
Final study report had been deferred to June 2017; however, long term extension discontinued in March 2014. Final study report planned for December 2014.
Based on the results of the Phase 1 protocol 20050252 (i.e., provided that a safe and tolerable dose of Panitumumab can be determined for children), Amgen will conduct a Phase 2 study to further assess the safety and to estimate the anti-tumor
To conduct an "Antibody Registry Study" that will enroll subjects who have received romiplostim and whose blood samples contain antibodies to either romiplostim or thrombopoietin.  The antibody assays will be performed by Amgen in response to s
Submit safety data assessing distant spread of toxin effects after multiple administrations of Xeomin (incobotulinumtoxinA), during a minimum period of 12 months, collected in at least 100 pediatric patients (ages 2-17 years). Approximately one
To develop a validated assay for identification of anti-product antibodies that neutralize the bioactivity of tbo-filgrastim.  The validation of the assay should include the sensitivity and specificity for detection of anti- tbo-filgrastim anti
Deferred pediatric Placebo-Controlled Efficacy and Safety Study under PREA for prophylaxis of headaches in adolescents ages 12 to 17 with chronic migraine. The study must include a prospective baseline observation period of at least 4 weeks fol
As of December 2013, 40 patients have been enrolled in the study.
To submit a separate subsection in the Periodic Safety Update Report (PSUR) containing the interim results of the enhanced pharmacovigilance monitoring plan for PML annually for ten years beginning in 2008.  This section will contain the ongoin
To conduct a multi-center, dose-finding study to determine the optimum starting dose of intravenously administered methoxy polyethylene glycol-epoetin beta when used for the maintenance treatment of anemia in pediatric patients ages 5 to 17 yea
Final Study report is deferred to August 2017.
Conduct a deferred pediatric study under PREA to determine whether, compared to placebo, administration of Palifermin decreases the incidence and duration of severe oral mucositis and related sequelae experienced by patients age 3-16 with hemat
January 5, 2009, FDA replaced PMC-1 with two new PMCs: PMC 38-1 and PMC 38-2. (PMC 38-1 was fulfilled May 30, 3013).
Conduct a prospective, multi-center registry including 5000 adult psoriasis patients treated with Humira in the United States.  This registry will characterize and assess the incidence of serious adverse events (including serious infections, tu
To submit a protocol for review for a prospectively and actively enrolled pregnancy registry that will collect information assessing pregnancy complications and birth outcomes in women with breast cancer exposed to a Herceptin-containing regime
A randomized, double-blind, placebo-controlled trial to evaluate the effect of long-term treatment with Contrave (naltrexone hydrochloride/bupropion hydrochloride) extended-release tablets on the incidence of major adverse cardiovascular events
Contrave® (Naltrexone Hydrochloride and Bupropion Hydrochloride)
Enhanced pharmacovigilance program for reports of malignancy in pediatric, adolescent, and young adult (< 30 years of age) patients treated with Humira (adalimumab), for a period of up to 10 years after this notification to collect data that wi
Long-term pediatric study in patients ? 10 years to ? 17 years of age to evaluate the safety and efficacy of BOTOX in the treatment of urinary incontinence due to detrusor overactivity associated with a neurologic condition (e.g. spina bifida o
Study 191622-121 is ongoing with 39 subjects currently enrolled and 12 subjects having completed the study. Allergan plans to enroll 100 subjects.
Conduct studies to evaluate the safety and efficacy of ustekinumab in pediatric subjects with plaque psoriasis.
Evaluate long-term safety and efficacy data in a Clinical Surveillance Program (CSP) of patients being treated with Galsulfase.  Detailed clinical status information will be collected at study entry and on an annual basis for at least 15 years.
Provide clinical information from a 1-year (minimum) clinical trial evaluating the adverse effects, if any, on the corneal endothelium following administration of aflibercept.
To conduct an assessment of anti-drug antibody (ADA) response and neutralizing ADA responses to ipilimumab with a validated assay (required in PMR 2 and 3) capable of sensitively detecting ADA responses in the presence of ipilimumab levels that
Assess data anticipated from adults in protocol "TYSABRI Observational Study in Safety in CD (Crohn's disease)" (Postmarketing Commitment Number 4, below) and establish a pediatric study plan that incorporates these new data.  These data must b
To identify further genetic determinants of immune-mediated adverse events caused by ipilimumab. DNA samples from the required postmarketing study comparing 3 mg/kg vs. 10 mg/kg ipilimumab monotherapy will be used to conduct genome-wide associa
Conduct a juvenile animal toxicology study of 3 months duration in an appropriate species before initiation of the pediatric  studies in patients 5 to 17 years of age.
The study is proceeding according to the original schedule.
Develop and validate an improved immunogenicity assay for detecting neutralizing antibodies to Galsulfase.
Submit safety data assessing distant spread of toxin effects after multiple administrations off Myobloc (botulinum toxin Type B), during a minimum period of 12 months, collected in at least 100 pediatric patients (ages 2-17 years) and 100 adult
The protocols for the clinical safety studies have not yet been finalized by the  Sponsor.
Submit safety data assessing distant spread of toxin effects after multiple administrations of Dysport (abobotulinumtoxinA), during a minimum period of 12 months, collected in at least 100 adult patients (approximately half upper, and half lowe
Obtain preliminary safety and activity data and to characterize the pharmacokinetics of Bevacizumab in pediatric patients in Study AVF2117s, a Phase 1, dose-escalation trial, enrolling up to 24 children with relapsed or refractory solid tumors
Evaluate long-term safety and activity data in a registry study of patients being treated with Laronidase.  Among other things, the registry data will be used to evaluate the effect of Laronidase on pregnancy and lactation, and potential effect
Conduct a randomized, controlled clinical trial in patients with lupus nephritis to evaluate the efficacy and safety of Benlysta (belimumab).
The study completion and final report due dates have not been met.
Submit a final report for the long-term, open-label, continuation trial C1066.
To conduct a study to assess for the immunogenicity of Myalept (metreleptin) in a relevant number of patients receiving metreleptin. The study should include testing for anti-metreleptin and anti-native human leptin binding antibodies at times
A long-term prospective observational study (product exposure registry) of patients treated with Myalept (metreleptin), regardless of indication, to evaluate serious risks related to the use of Myalept (metreleptin), by indication, including: f
Conduct an observational prospective pharmacoepidemiology cohort study (Protocol IM101045B) to assess the short and long-term risk of malignancies and infection in patients with RA treated with Abatacept in comparison to other DMARDs within an
Enrollment completion/observation completion dates, 6/2009 & 6/2014 missed. Enrollment target 5000, as of 6/30/2015, 1370 enrolled. FSR due 12/31/16.
To evaluate the safety and tolerability of an alternate Herceptin (trastuzumab) dosing regimen that ensures that all patients, inclusive of patients with a Herceptin Cmin of ? 12 mcg/mL on Cycle 1 Day 21 after an initial dose of 8 mg/kg, achiev
The trial was terminated on the basis of futility. The final report is expected by March 31, 2016.
To conduct a study to evaluate the impact of Oprelvekin dosing on QT interval prolongation.  The study should include up to 60 patients with cancer for whom Oprelvekin is indicated, who will receive Oprelvekin as subcutaneous injections at the
Conduct a study to evaluate pediatric pharmacokinetics (PK), safety and antiviral activity of once daily darunavir and cobicistat (DRV/COBI) combined with a background regimen in HIV-1 infected pediatric subjects. Subjects receiving DRV/COBI sh
Enrollment for the study is currently ongoing.
A clinical trial in healthy subjects evaluating the effect of cobicistat coadministered with darunavir at steady state on the pharmacokinetics of atorvastatin.
Establish a Pregnancy Registry to collect and analyze information for ten years on pregnancy complications and birth outcomes in women with breast cancer exposed to a pertuzumabcontaining regimen within 6 months of conception or during pregnanc
A randomized, double blind, placebo-controlled trial evaluating the effect of Tanzeum (albiglutide) on the incidence of major adverse cardiovascular events (MACE) in subjects with type 2 diabetes mellitus. The primary objective of the trial sho
A study evaluating gallbladder ejection fractions in albiglutide treated subjects to further characterize the effect of albiglutide on gallbladder motility.
Conduct a PK/PD study in adolescents ages > or equal to 13 years to 17 years with moderate to severe symptoms of primary Restless Legs Syndrome. Using information from this PK study, conduct a clinical trial to assess the efficacy and safety of
The final protocol milestone was missed because the protocol has not been agreed upon.
Following completion of protocol ALID 02307, patients will be encouraged to enroll in the ongoing MPS I Registry.  BioMarin is required to collect available mortality, immunogenicity, allergic reactions, and growth and development data on this
To conduct a prospective, observational study in at least 2000 subjects with Crohn's disease who are receiving TYSABRI, with each subject followed for at least five years, by completing protocol, "TYSABRI Observational Study in Safety in CD (Cr
Deferred pediatric efficacy study under PREA for the treatment of upper limb spasticity, to decrease the severity of increased muscle tone in the elbow flexors, wrist flexors and finger flexors in pediatric patients ages 2 years through 16 year
Original Final Report Due Date: 01/15/2016; Deferral Extension granted per FDA letter dated 11/23/2016.
Conduct ado-trastuzumab emtansine exposure-response analyses for progression-free survival, final overall survival, and safety utilizing data from trial BO25734/TDM4997 (TH3RESA).  The results of the exposure-response analyses from both TH3RESA
A pharmacokinetic and safety study of tocilizumab (TCZ) in patients less than 2 years old with active systemic juvenile idiopathic arthritis (sJIA)
Deferral Extension granted in FDA letter dated August 15, 2014. Current enrollment, 7 patients.
An observational safety study enrolling 500 patients treated with Krystexxa (pegloticase) for one year duration.  Patients enrolled should have hyperuricemia and gout and be refractory to standard uric acid-lowering therapies (e.g., allopurinol
Centocor agrees to conduct a prospective, multi-center registry including 4000 adult psoriasis patients treated with commercial REMICADE in the United States.  This registry will characterize and assess the incidence of serious adverse events (
Establish a Pregnancy Registry to collect and analyze information for 10 years on pregnancy complications and birth outcomes in women with breast cancer exposed to ado-trastuzumab-emtansine within 6 months of conception or during pregnancy. Sub
A study to analyze samples from the Pediatric IBD registry (DEVELOP) and PMR 3 to determine the presence of ADA using the new assay developed in PMC 5.
A long-term safety study in 400 pediatric patients 2-17 years of age with polyarticular JIA (pJIA) treated with tocilizumab to evaluate for the risk of malignancies, serious infections, gastrointestinal perforation, and effects on growth. The s
A juvenile rat toxicology study is required to identify the unexpected serious risk of adverse effects on postnatal growth and development. The study should utilize animals of an age range and stage(s) of development that are comparable to the
Analyze anti-elosulfase alfa neutralizing antibody titers in patient samples obtained in the completed MOR-004 trial.
Conduct a prospective registry of belatacept use in US adult kidney-only transplant recipients to determine the incidence rates of post-transplant lymphoproliferative disorder PTLD, PTLD in the central nervous system (CNS PTLD), and progressive
A study to measure GC-C mRNA levels in duodenal and colonic tissue obtained from children ages 0 to 6 years of age.
Conduct a randomized, double blind, multicenter, comparative study to establish the safety and tolerability profile of ceftolozane/tazobactam compared to that of meropenem in hospitalized children from birth to <18 years with cUTI. The dose for
Zerbaxa® (Ceftolozane Sulfate and Tazobactam Sodium)
Provide data analyses from the Pregnancy Research Initiative (study C0168T71).
A post-marketing, observational epidemiologic study comparing MOVANTIK (naloxegol) to other treatments of opioid induced constipation in patients with chronic non-cancer pain. The study’s primary outcome is a composite of major adverse cardio
An in vitro study to evaluate the time-dependent/mechanism-based inhibition potential of naloxegol on the hepatic CYP2C8 enzyme.
Deferred pediatric long-term safety study (minimum 12 months) under PREA for the treatment of upper limb spasticity in pediatric patients ages 2 years through 16 years 11 months. The doses evaluated must be at least as high as those shown effec
An observational study registry in adult patients with moderately to severely active RA that would assess the longer term risks of serious infections, malignancies that have been reported with TNF  blocker therapy, as well as the longer term ri
During the conduct of the required postmarketing trial comparing 3mg/kg vs. 10mg/kg ipilimumab monotherapy (PMR 6), you will obtain comprehensive baseline DNA sample acquisition (? 95% of ITT) and conduct pharmacogenomic association analyses to
Enroll 4,000 Stelara¿ (ustekinumab)-treated subjects into the Psoriasis Longitudinal Assessment and Registry, (PSOLAR) and follow for 8 years from the time of enrollment.  Subjects will be followed for the occurrence of serious infection, tube
Enhanced pharmacovigilance program for reports of malignancy in pediatric, adolescent, and young adult (< 30 years of age) patients treated with Enbrel (etanercept), for a period of up to 10 years after this notification to collect data that wi
A long-term safety study in 100 pediatric patients 2 to 17 years of age with systemic JIA (SJIA) treated with canakinumab to evaluate for the risks of serious infections, neutropenia, thrombocytopenia, severe injection site reactions, and MAS.
Evaluate the occurrence of serious infections associated with administration of a prophylactic immune tolerance regimen in a cohort of Morquio A syndrome patients treated with Vimizim (elosulfase alfa) who are at high risk of developing persist
A study in inflammatory bowel disease (IBD) patients treated with Humira (adalimumab) in which you will bank tissue or blood samples (as appropriate) and then analyze them to identify genetic mutations and other biomarkers that predispose these
Conduct a randomized, placebo-controlled, blinded, multicenter study of the induction and maintenance of clinical response and remission by Entyvio (vedolizumab) in pediatric patients 5 to 17 years of age with moderately to severely active ulce
Conduct a milk-only lactation study in lactating women receiving vedolizumab therapeutically to assess concentrations of vedolizumab in breast milk using a validated assay in order to appropriately inform the Nursing Mother’s subsection of la
Conduct a randomized, placebo-controlled, blinded, multicenter study of the induction and maintenance of clinical response and remission by Entyvio (vedolizumab) in pediatric patients 6 to 17 years of age with moderately to severely active Croh
Submit safety data assessing distant spread of toxin effects after multiple administrations of Dysport (abobotulinumtoxinA), during a minimum period of 12 months, collected in at least 100 pediatric patients (ages 2-17 years) (approximately hal
The final report milestone was missed because one of the studies contributing to the results is ongoing because of difficulty with enrollment. FDA determined that the applicant demonstrated good cause for the delay.
Randomized, double-blind, adequate and well controlled, multiple fixed-dose, parallel group clinical trial of Xeomin (incobotulinumtoxinA) in botulinum toxin-naive adults with lower extremity spasticity. The minimum duration of the trial should
Conduct a randomized, controlled clinical trial to evaluate the efficacy and safety of Benlysta (belimumab) in African-American patients with SLE.
Conduct a one-year, multi-center, randomized, double-blind placebo-controlled trial to evaluate the efficacy, safety and pharmacokinetics of adalimumab in pediatric patients 5 to 17 years of age with moderately to severely active ulcerative col
To determine the approximate percent of potential impurities derived from the E. coli cell line used to manufacture metreleptin that are detected by the ELISA to assess for host cell proteins (HCP) in metreleptin drug substance using a sensitiv
Utilizing a validated AAA assay as described in PMR #3 above, you should measure and analyze the immunogenicity profile based on post-dose patient samples from completed study M10-223, the trial conducted under PMR #5, the trial conducted under
Pediatric study in patients  10 years to 17 years of age to evaluate the safety and efficacy of BOTOX in the treatment of urinary incontinence due to detrusor overactivity associated with a neurologic condition (e.g. spina bifida or spinal cord
Study 191622-120 is ongoing with 61 subjects currently enrolled and 37 subjects having completed the study. Allergan plans to enroll 132 subjects.
Conduct a clinical trial to further assess the cardiac safety of neoadjuvant anthracycline/taxane-based chemotherapy regimens when administered in combination with neoadjuvant pertuzumab and trastuzumab in patients with locally advanced, inflam
Randomized, double-blind, adequate and well controlled, parallel group, clinical trial of Xeomin (incobotulinumtoxinA) in botulinum toxin-naive adults with blepharospasm. You should propose a method to actively monitor for adverse events relate
Randomized, double-blind, adequately controlled, multiple fixed dose, parallel group clinical trial of Botox (onabotulinum toxin type A) in botulinum toxin-naïve children age 2- 17 years with lower extremity spasticity.  The minimum duration o
Evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of darunavir and cobicistat fixed dose combination (FDC) age-appropriate formulation in HIV-infected pediatric subjects 3 years to less than 6 years of age and weighing at
Evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of darunavir and cobicistat fixed-dose combination (FDC) tablets in HIV-infected pediatric subjects 12 years to less than 18 years of age. The safety and antiviral activit
Evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of atazanavir and cobicistat fixed-dose combination (FDC) age-appropriate formulation in HIV-infected pediatric subjects 3 years to less than 6 years of age. The safety an
Evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of atazanavir and cobicistat fixed-dose combination (FDC) age-appropriate formulation in HIV-infected pediatric subjects 6 years to less than 12 years of age The safety an
Evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of atazanavir and cobicistat fixed-dose combination (FDC) age-appropriate formulation in HIV-infected pediatric subjects 12 years to less than 18 years of age. The safety
Conduct an adequate leachable safety assessment for your freeflex® bag container closure system. This assessment must include leachable data from longterm stability studies testing at least three batches at multiple time points over the shelf-
Conduct the deferred pediatric study to assess the pharmacokinetics, safety/tolerability, and antiviral activity of tenofovir alafenamide in HBV infected subjects 12 to less than 18 years of age, followed by a rollover to a long-term, open-labe
Conduct the deferred pediatric study to access the pharmacokinetics, safety/tolerability, and antiviral activity of tenofovir alafenamide in HBV infected subjects 2 to less than 12 years of age, followed by a rollover to a long-term, open-label
A postmarketing study that will allow you to test for and control the levels of the following elemental impurities in your product: [...] that are present during the manufacturing process and have the potential to contaminate your product. As p
A prospective, multi-center, long-term, observational study of ulcerative colitis patients treated with Simponi (golimumab) in a routine clinical setting, to assess the long-term safety of Simponi (golimumab). The study’s primary outcome shou
Conduct a study to evaluate the pharmacokinetics of Simponi (golimumab) in pediatric patients 5 to 17 years of age with moderately to severely active ulcerative colitis. Pharmacokinetic measurements should be conducted for exposure-response ana
Conduct a study to evaluate the effectiveness and safety of Simponi (golimumab) in pediatric patients 5 to 17 years of age with moderately to severely active ulcerative colitis. The study should be designed to establish that the dose regimen(s)
Conduct a pharmacokinetic study in healthy volunteers to evaluate the effects of administration methods on pharmacokinetics of cysteamine. This study will compare the pharmacokinetics of cysteamine after administration of Procysbi (cysteamine b
Confirm the efficacy and safety of Trulance (plecanatide) treatment in pediatric patients with chronic idiopathic constipation (CIC) who are 2 years to less than 6 years of age by performing a randomized, double-blind, placebo-controlled, paral
Assess the long-term safety of Trulance (plecanatide) in pediatric patients with chronic idiopathic constipation (CIC) who are 2 years to less than 18 years of age and have completed a confirmatory efficacy and safety study with plecanatide.
Submit and analyze quarterly reports until March 2017 of complaints about preparation and use of Treanda Injection (solution) and Treanda for Injection (lyophilized powder).
Conduct a study to evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response) of elbasvir and grazoprevir in pediatric subjects 3 to 17 years of age with chronic hepatitis C infection.
Collect and analyze long-term safety data from pediatric subjects 3 to 17 years of age enrolled in the pediatric elbasvir and grazoprevir safety, pharmacokinetic and efficacy study. Data should be collected for at least 3 years following the en
Conduct site-directed mutant phenotype analyses of elbasvir against HCV replicons carrying the following NS5A substitutions: K24R (GT1a), H54Y (GT4d), E62D (GT1a), D427N (GT1a). Include cross-resistance analyses with approved NS5A inhibitors.
Conduct site-directed mutant phenotype analyses of grazoprevir against HCV replicons carrying the following NS3 substitutions: I48A/V (GT1a), T185S (GT1a/GT1b), E357G/K (GT1a). Include cross-resistance analyses with approved NS3/4A inhibitors.
Conduct a trial in hepatitis C virus genotype 1a infected subjects with at least one baseline NS5A polymorphism at amino acid position 28, 30, 31, or 93 to evaluate if treatment with elbasvir/grazoprevir and ribavirin for at least 16 weeks redu
Evaluate the effect of SLCO1B1 genotype on grazoprevir pharmacokinetics (PK) and response to elbasvir/grazoprevir treatment in patients with chronic hepatitis C virus infection. To evaluate this effect, either conduct a prospective clinical tri
Submit the final report and datasets, including the SVR24 data, for Phase 3 Trial 068 (C EDGE TE).
Submit the final report and datasets, including the SVR24 data, for Phase 2 Trial 048 (C-SALVAGE).
Submit the final report and datasets, including the SVR24 data, for Phase 3 Trial 061 (C-EDGE CO-INFECTION).
Perform genotypic (also phenotypic if qualified) resistance analysis of baseline virus samples from all HBeAg-positive nucleos(t)ide reverse transcriptase inhibitor-experienced subjects and of Week-48 virus samples from all evaluable subjects i
Conduct a field study to evaluate the efficacy and safety of ciprofloxacin in the event of an attack with the intentional release of Y. pestis in the United States.
Deferred pediatric study under PREA for the treatment of major depressive disorder in pediatric patients aged 7 to 17. Conduct a study to obtain data on the efficacy and safety of desvenlafaxine in the relevant pediatric population.
Conduct your deferred pediatric study in HIV-1 infected patients 4 weeks to less than 6 years to assess the pharmacokinetics, safety and tolerability, and antiviral activity of an age-appropriate formulation of emtricitabine and tenofovir alafe
Descovy® (Emtricitabine and Tenofovir Alafenamide)
Conduct your deferred pediatric study in HIV-1 infected, virologically suppressed patients 6 years to less than 12 years switching from other nucleoside reverse transcriptase inhibitors (NRTIs) to assess the pharmacokinetics, safety, tolerabili
A prospective, controlled, non-interventional, long-term cohort study that follows a series of cohorts consisting of new users of Natazia (estradiol valerate and estradiol valerate/dienogest) tablets and new users of oral contraceptives contain
Natazia® (Estradiol Valerate; and Estradiol Valerate and Dienogest)
Develop and validate a sensitive and precise assay for the detection of anti-plecanatide antibodies (ADA), including IgM, IgG, and IgA, that may be present in the serum at the time of patient sampling.
Develop and validate assays to evaluate the cross reactivity of anti-plecanatide antibodies to guanylin and uroguanylin.
Develop and validate an assay to evaluate the neutralizing capacity of ADAs detected in the patient samples taking Trulance (plecanatide).
A study to characterize guanylate cyclase-C (G-CC) mRNA expression in duodenal and colonic mucosal biopsies in pediatric patients ages 0 to 6 years undergoing diagnostic gastrointestinal endoscopies as part of their medical care.
Assess development of anti-drug antibody (ADA) responses in patient samples using the immunogenicity serum samples collected in the plecanatide studies (SP304203-00 and SP304203-03 and SP304203-01). Validated assays capable of sensitively and a
Use the validated cross reactivity assays developed under PMR 3117-8 to test the ADA positive samples detected under PMR 3117-11. Evaluate the relationships between cross reactivity status and the safety and efficacy of Trulance (plecanatide).
Use the validated neutralizing antibody assay developed under PMR 3117-9 to test the ADA positive samples detected under PMR 3117-11. Evaluate the relationships between neutralizing antibody status and the safety and efficacy of Trulance (pleca
Perform a milk-only lactation trial in lactating women who have received multiple, once daily, doses of Trulance (plecanatide) therapeutically to assess concentrations of plecanatide and its active metabolite in breast milk using a validated as
A multicenter, randomized, double-blind study evaluating the safety, efficacy and pharmacokinetics of Jublia (efinaconazole) topical solution, 10% versus vehicle in pediatric subjects ages 12 to less than 17 years with onychomycosis of the toen
A clinical trial to assess the risk of QT prolongation with Bunavail (buprenorphine and naloxone) buccal film.
Revised draft protocol submitted on 2/26/16, which is under review
Conduct a randomized Single-Blind, Multicenter Safety and Efficacy Study of Intravenous to Oral SIVEXTRO (tedizolid phosphate) and Intravenous to Oral Comparator for the Treatment of Acute Bacterial Skin and Skin Structure Infections in Pediatr
Original Final Report Due Date: 06/30/2017; Deferral Extension granted per FDA letter dated 01/10/2017.
Conduct a randomized, Single-Blind, Multicenter Safety and Efficacy Study of Intravenous to Oral SIVEXTRO (tedizolid phosphate) and Intravenous to Oral Comparator for the Treatment of Acute Bacterial Skin and Skin Structure Infections in Pediat
Deferral Extension Requested 07/26/2016.  Denied per FDA letter dated 01/10/2017.
Conduct an open-Label, Multicenter Study of 10-14 days IV SIVEXTRO (tedizolid phosphate) for hospital-acquired late onset sepsis in full term and preterm neonates and infants aged 5 days to < 3months.
Conduct a Phase 1 Single-Dose Safety and Pharmacokinetic Study of Oral and IV SIVEXTRO (tedizolid phosphate) in Patients 2 years to < 12 years of age.
Conduct a Phase 1 Single-Dose Safety and Pharmacokinetic Study of Oral and Intravenous SIVEXTRO (tedizolid phosphate) in Inpatients Under 2 Years Old.
Conduct US surveillance studies for five years from the date of marketing SIVEXTRO to determine if resistance to tedizolid has developed in those organisms specific to the indication in the label for ABSSSI.
An observational study of pediatric patients from birth to 3 years of age who are administered iothalamate meglumine for clinical evaluation, in order to assess their risk of developing hypothyroidism.
A 24-week, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of empagliflozin for the treatment of pediatric patients 10 to 17 years (inclusive) with type 2 diabetes mellitus as an add-on to metformin, follo
The final protocol milestone was missed because protocol finalization is dependent on the results of PMR 2755-1, for which the Final Report Submission date was extended. Deferral Extension Requested 05/15/2015. Denied per FDA letter dated 06/19
A study to evaluate empagliflozin toxicity in juvenile rats.
Per FDA letter dated 02/05/2016, this PMR has been fulfilled.
A randomized, double-blind, placebo-controlled trial evaluating the effect of empagliflozin on the incidence of major adverse cardiovascular events (MACE) in patients with type 2 diabetes mellitus. The primary objective of the trial should be t
Pediatric studies under PREA for the treatment of overactive bladder in pediatric patients for ages five to 11 years old and adolescents for ages 12 to 17 years old.
Original Final Report Due Date: 06/30/2015.  Per FDA letter dated 05/28/2014, Final Report due date extended to 06/30/2016.  Second Deferral Extension Granted per FDA letter dated 11/19/2014, Final Report due date extended to 8/17/2017. Study 9
Conduct a cell-culture 2-drug combination study to evaluate the anti-HBV activity of tenofovir alafenamide (TAF) in combination with sofosbuvir.
To evaluate potential tenofovir alafenamide (TAF) resistance pathways, sequence the baseline and Week 48 time-points (by population sequencing or NGS) for all evaluable subjects who had HBV DNA >69 IU/mL and provide a study report that includes
To evaluate potential tenofovir alafenamide (TAF) resistance pathways and provide a study report that includes resistance data analysis for evaluable samples at baseline, Week 48 and Week 96 and submit the fastq files and analyses for subjects
Provide a study report that includes resistance data analysis and submit the fastq files and analyses for subjects 8006-5282 and 8600-4558 who had HBV DNA titers at the last PCR assessment that were >159 IU/mL.
Phenotype Week-48 virus samples from Subjects 4296-5147 and 8758-5188 in the tenofovir alafenamide (TAF) group and Subjects 1507-4546 and 9035-4845 in the tenofovir disoproxil fumarate (TDF) group in Study GS-US-320-0110.
Submit the long-term efficacy, safety and antiviral activity data for Studies GSUS-320-0108 and GS-US-320-0110. Include data and analyses for the entire study population through Week 144.
Provide the results of an analytic validation study for the assay used to identify patients with PD-L1 positive and PD-L1 negative SCCHN in Study CA2090141 to inform product labeling for the device and for nivolumab.
Phase 2, multicenter study to evaluate the safety, efficacy, and pharmacokinetics of belimumab plus background standard therapy in 70 pediatric subjects ages 5 years to 17 years of age with active systemic lupus erythematosus (SLE).
A post-marketing, observational epidemiologic study comparing Relistor (methylnaltrexone bromide) to other treatments of opioid induced constipation in patients with chronic non-cancer pain. The study’s primary outcome is a composite of major
Conduct epidemiologic investigations to address whether the properties intended to deter misuse and abuse of EMBEDA (morphine sulfate and naltrexone hydrochloride extended-release capsules) actually result in a significant and meaningful decrea
Conduct a clinical trial to evaluate the pharmacokinetics, safety, and antiviral activity of peramivir administration in pediatric subjects with acute uncomplicated influenza infection from birth to less than 18 years of age. Include characteri
Reporting the enrollment in the study is ongoing with 18 patients randomized and 15 patients treated as of 10 February 2016.
Analyze and submit the remainder of the clinical resistance data that were not included with the NDA. These include both the HA and NA data for trials BCX1812-201, BCX1812-211, and BCX1812-311.
Conduct a study to determine the cross-resistance to oseltamivir and zanamivir for all of the HA peramivir resistance substitutions that have yet to be evaluated (A/H1N1 HA D129S, R208K; A/H3N2 HA G78D, K189E; B HA T139N, G141E, R162M, D195N, T
A postmarketing study that will allow you to test for and control the levels of the following elemental impurities in your product: [...] present during the manufacturing process and have the potential to contaminate your product. As part of yo
The supplement was approved on 3/22/16. The PMR is delayed because final study report date for all NDAs specified in this PMR was missed.
Normosol™ R and Dextrose (Dextrose and Multiple Electrolytes Type 1)
Aminosyn™ II (Amino Acid and Electrolytes)
Normosol™ M and Dextrose (Dextrose and Multiple Electrolytes Type 1)
Potassium Chloride and Sodium Chloride in Dextrose
Isolyte® S in EXCEL® Plastic Container (Dextrose and Multiple Electrolytes)
Normosol®-R and Normosol®-R pH 7.4 (Multiple Electrolytes Type 1)
Potassium Chloride in Dextrose in Lactated Ringer's
Dextrose and Ringer's® in Plastic Container (Dextrose and Lactated Ringer's)
Magnesium Chloride and Potassium in Dextrose
SODIUM LACTATE INJECTION IN PLASTIC VIAL
Assessment of efficacy and safety of OraVerse in patients from 3-6 years of age. This study should be a randomized, sham-injection controlled, double-blinded study comparing the times to return of normal sensation and normal function following
Per FDA letter dated 03/18/2016, this PMR has been fulfilled.
Submit the complete final report and data from ongoing trial GO28667, a randomized, Phase 3 trial comparing VENCLEXTA (venetoclax) and rituximab with bendamustine and rituximab in patients with relapsed or refractory chronic lymphocytic leukemi
Evaluate the effect of hepatic impairment on the pharmacokinetics and safety of VENCLEXTA (venetoclax)compared to subjects with normal hepatic function. Submit a complete final report with all supporting datasets for trial M15-342 entitled, “
Evaluate the effect of VENCLEXTA (venetoclax) co-administration on pharmacokinetics of a probe substrate of P-gp. Submit a complete final trial report with all supporting datasets.
Assessment of Amjevita (adalimumab-atto) for the treatment of Polyarticular juvenile idiopathic arthritis (pJIA) in patients 2 years to less than 4 years of age.
Assessment of Amjevita (adalimumab-atto) for the treatment of pediatric Crohn’s disease in pediatric patients 6 years to 17 years of age.
Assessment of Amjevita (adalimumab-atto) for the treatment of pediatric ulcerative colitis in pediatric patients 5 years to 17 years of age.
Develop a presentation that can be used to accurately administer Amjevita (adalimumab-atto) to pediatric patients who weigh less than 15 kg.
Deferred pediatric study under PREA investigating ORAVIG safety, pharmacokinetics, efficacy and compliance with use instructions in patients with oropharyngeal candidiasis ages > 5 to < or = 17 years.
Final report was due on March 31, 2014. The Sponsor states that since the ownership of this NDA changed so many times, the study is not done.
Evaluate the impact of peramivir resistance-associated substitutions in hemagglutinin (HA) on the effectiveness of influenza vaccine in cell culture assays: • Titrate the neutralization and hemagglutinin inhibition activity of the serum sampl
Submit clinical data from an adequate number of subjects to characterize the effectiveness of peramivir administration in patients with acute uncomplicated influenza B virus infection. These data may be collected from the pediatric study requir
The final analysis plan submission due date was 06/30/15
Conduct a clinical trial to evaluate the pharmacokinetics, safety and antiviral activity of peramivir administration in a predominantly ambulatory setting in elderly subjects aged 65 years or older with influenza infection.
Conduct a clinical trial to evaluate the pharmacokinetics, safety and antiviral activity of peramivir administration in a predominantly ambulatory setting in subjects with influenza infection at higher risk for influenza complications, as defin
Open-label long term extension study for PMR 2099-#3 (A prospective, randomized, controlled, double- blind, efficacy and safety study of eslicarbazepine acetate in children ages 12 years to <18 years for the adjunctive the treatment of partial
A prospective, randomized, controlled, double-blind, efficacy and safety study of eslicarbazepine acetate in children ages 2 years to < 12 years for the adjunctive treatment of partial onset seizures. The primary efficacy endpoint during the co
Open-label long term extension study for PMR 2099-5 (A prospective, randomized, controlled, double-blind, efficacy and safety study of eslicarbazepine acetate in children ages 2 years to < 12 years for the adjunctive treatment of partial onset
A prospective, randomized, controlled, double-blind, efficacy and safety study of eslicarbazepine acetate for the adjunctive treatment of partial onset seizures in children ages 1 month to < 4 years. The primary efficacy endpoint must examine s
Long term extension study for PMR 2099-7 (A prospective, randomized, controlled, double-blind, efficacy and safety study of eslicarbazepine acetate for the adjunctive treatment of partial onset seizures in children ages 1 month to < 4 years). S
An ex vivo study to determine whether eslicarbazepine interferes with assays for free T3 and T4 as well as total T3, T4, and TSH. Collect blood samples from 30 subjects who have taken a daily dose of at least 1200 mg eslicarbazepine acetate for
The study completion milestone was missed due to difficulty in enrolling subjects. Additional sites currently being identified.
A study based on routine postmarketing safety surveillance, pharmacovigilance and clinical trial reports will characterize clinical and genomic risk factors associated with the development of serious dermatologic reactions in eslicarbazepine ac
The study completion milestone was missed due to delayed submission of final protocol and ongoing enrollment.
A prospective human physical dependence trial in healthy volunteers in which subjects are titrated to 800 mg of eslicarbazepine acetate and maintained at this dose for four weeks. At the end of the treatment, the drug should be abruptly withdra
The final protocol milestone was missed due to delayed submission of draft protocols. Draft protocols under FDA review
Deferred pediatric study under PREA for the treatment of schizophrenia in pediatric patients aged 13 to 17. Conduct a study to obtain pharmacokinetic, safety, and tolerability data and provide information pertinent to dosing brexpiprazole in th
Original Final Report Due Date: 11/30/2016; Deferral Extension granted per FDA letter dated 10/08/2016.
To evaluate the safety of palifermin in pediatric hematopoietic stem cell transplant patients in the prospective cohort study 20050187. This will be a component of the phase 4 prospective cohort study using the available Center for Internationa
The bi-annual interim report was submitted June 27, 2014. Trial completion due by September 2019
A clinical pharmacology (Part A) followed by a 52-week randomized, doubleblind and placebo-controlled pediatric study (Part B) under PREA to evaluate the pharmacokinetics, safety, and efficacy of Contrave (naltrexone/bupropion) for the treatmen
The protocol finalization milestone was missed because of ongoing discussion with FDA.
A clinical pharmacology (Part A) followed by a 52-week randomized, double-blind and placebo-controlled pediatric study (Part B) under PREA to evaluate the pharmacokinetics, safety, and efficacy of Contrave (naltrexone/bupropion) for the treatme
A thorough QT trial designed to rule out small changes in QTc interval (i.e.,upper bound of 90% confidence interval excludes 10 ms), as defined by ICH E14 guidance.
Conduct a study to evaluate the efficacy and long-term safety of mometasone furoate/formoterol fumarate combination MDI (Dulera) and mometasone furoate MDI (Asmanex HFA) in children 5 to 11 years of age.
The study has not been initiated, but does not meet the criterion for delayed. Protocol submitted 8/14/15.
Six-month oral toxicology study of methacrylic acid copolymer, [...] , in rat. The methacrylic acid copolymer, [...] , should be the same as the excipient in the to-be-marketed product.
Oral absorption study of radiolabeled methacrylic acid copolymer, [...], in rat. The methacrylic acid copolymer, [...], should be the same as the excipient in the to-be-marketed product.
Conduct a multicenter, evaluator blinded, randomized comparator study designed to assess the safety, efficacy and PK of three age dependent doses of IV daptomycin administered for up to 14 days in pediatric patients aged 1 to 17 years, inclusiv
Deferral Extension Requested 10/07/2015.  Denied per FDA letter dated 03/02/2016.
Complete a clinical pharmacokinetic trial to determine the appropriate dose of cobimetinib in patients with hepatic impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics in Patients with Impaired Hepatic Funct
Provide an integrated safety analyses from an adequate number of randomized controlled clinical trials using cobimetinib to identify and characterize the risk of retinal pigmented epithelial detachments (RPED) and subsequent sequelae, including
Submit the clinical report at the time of the final analysis of Trial GO28141, A Phase III, Double-Blind, Placebo-Controlled Study of Vemurafenib Versus Vemurafenib Plus Cobimetinib (GDC-0973) in Previously Untreated BRAFV600-Mutation Positive
Conduct a study to evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response) of ledipasvir/sofosbuvir in pediatric subjects 3 through 17 years of age with chronic hepatitis C.
Enrollment for the study is currently ongoing
Conduct a study to evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response) of ledipasvir/sofosbuvir in pediatric subjects 3 through 17 years of age with chronic hepatitis C
Conduct a study to characterize the cell culture antiviral activity of ledipisvir against representative HCV subtype 4b and 4r isolates, including those with amino acid variability (relative to subtypes 4a and 4d) at NS5A positions 28, 30, 31 a
Conduct and submit the results of at least one multicenter, randomized clinical trial establishing the superiority of osimertinib over available therapy as determined by progression-free or overall survival in patients with metastatic epidermal
A clinical trial in healthy subjects evaluating the effect of cobicistat coadministered with darunavir at steady state on the pharmacokinetics of rosuvastatin.
A clinical trial in healthy subjects evaluating the effect of cobicistat coadministered with darunavir at steady state on the pharmacokinetics of the estrogen and progestin components of a combined oral contraceptive.
A 56-week randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of Saxenda for the treatment of obesity in pediatric patients ages 12 to 17 (inclusive).
The Final Protocol Submission milestone was missed because of ongoing discussions with FDA regarding study design. FDA determined that the applicant demonstrated sufficient justification for the delay.
A clinical pharmacology study to assess pharmacokinetic and pharmacodynamics parameters of Saxenda in obese pediatric patients ages 7 to 11 years (inclusive).
A 56-week randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of Saxenda for the treatment of obesity in pediatric patients ages 7 to 11 (inclusive). The trial may not be initiated until results from the Saxen
A medullary thyroid carcinoma registry-based case series of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction o
The Final Report Submission milestone was missed because trial initiation was delayed due to protocol discussion with FDA and additional analyses were required that were expected to lengthen the report preparation time between trial completion
To assess the risk of breast cancer associated with liraglutide in the LEADER (Liraglutide Effect and Action in Diabetes: Evaluation of Cardiovascular Outcome Results) cardiovascular outcomes trial. To assess this risk, collect information on b
To assess the risk of breast cancer associated with liraglutide in Trial 1839. To assess this risk, collect information on baseline cancer risk and potential confounders for all identified cases of breast cancer in the trial, including (but not
A study evaluating gallbladder ejection fractions in liraglutide-treated subjects to further characterize the effect of liraglutide on gallbladder motility.
An open-label pharmacokinetic and safety study or studies of an age-appropriate formulation of Dyloject (diclofenac sodium) Injection in pediatric patients 2 to less than 17 years of age with acute pain.
JAVELIN PHARMACEUTICALS INC A WHOLLY OWNED SUDSIDIARY OF HOSPIRA INC
A pharmacokinetic, safety, and efficacy study or studies of an age-appropriate formulation of Dyloject (diclofenac sodium) Injection in pediatric patients 1 to less than 2 years of age with acute pain. Conduct the study or studies after the juv
A juvenile animal study to evaluate the general toxicology of the Dyloject (diclofenac sodium) Injection pediatric formulation to support the safe use of the pediatric formulation prior to initiation of the clinical study in pediatric patients
Deferred pharmacokinetics (PK) and safety study of Caldolor (ibuprofen) Injection for reduction in fever, or management of mild-to-moderate pain and management of moderate to severe pain as an adjunct to opioid analgesics, in pediatric patients
Conduct a 12 week, randomized, open-label, pharmacokinetic and pharmacodynamics study of Nucala (mepolizumab) in pediatric patients with asthma 6 years to 11 years of age (Part A of Study 200363).
36 subjects have been enrolled, accrual completed on 7/14/16.
Conduct a 12 month long-term safety and pharmacodynamics extension study of Nucala (mepolizumab) in pediatric patients with asthma 6 years to 11 years of age (Part B of Study 200363).
38 subjects have been enrolled, accrual completed on 7/14/16.
Conduct your deferred pediatric study in HIV-infected patients 6 years to less than 12 years to assess the pharmacokinetics, safety and tolerability, and antiviral activity of age-appropriate doses of elvitegravir, cobicistat, emtricitabine, an
Plans to submit final study report per PMR timeline
Genvoya® (Elvitegravir, Cobicistat, Emtricitabine, and Tenofovir Alafenamide)
Submit the long-term safety and antiviral activity data for Study GS-US-292-0106. Include data and analyses for the entire study population through Week 48 and for all subjects enrolled in the extension phase through 96 weeks of GENVOYA® dosin
Using appropriate in vitro approaches (including, but not limited to, patch clamp studies of L-type and T-type calcium channels and transporter phenotyping), evaluate the potential mechanism of both pharmacodynamic and pharmacokinetic interacti
Evaluate the effect of hepatic impairment on the pharmacokinetics and safety of Folotyn (pralatrexate). Submit a complete final report with all supporting datasets.
An observational study of pediatric patients from birth to 3 years of age who are administered iohexol for clinical evaluation, in order to assess their risk of developing hypothyroidism.
An observational study of pediatric patients from birth to 3 years of age who are administered diatrizoate meglumine; diatrizoate sodium for clinical evaluation, in order to assess their risk of developing hypothyroidism.
MD-76R (Diatrizoate Meglumine and Diatrizoate Sodium)
An observational study of pediatric patients from birth to 3 years of age who are administered ioversol for clinical evaluation, in order to assess their risk of developing hypothyroidism.
Optiray™ 240, Optiray™ 300, Optiray™ 320, Optiray™ 350
Optiray™ 300 PBP, Optiray™ 320 PBP, Optiray™ 350 PBP (Ioversol)
An observational study of pediatric patients from birth to 3 years of age who are administered ioxilan for clinical evaluation, in order to assess their risk of developing hypothyroidism.
An observational study of pediatric patients from birth to 3 years of age who are administered iopamidol for clinical evaluation, in order to assess their risk of developing hypothyroidism.
An 8-week GLP toxicology study with fertility evaluation in neonatal rats treated with netupitant alone.
The final study report was submitted to the FDA on 3/10/16.
Akynzeo® (Netupitant and Palonosetron Hydrochloride)
A PK/PD dose finding study of netupitant to characterize the netupitant PK/PD relationship for complete response in the delayed phase following oral administration of a single dose of netupitant given concomitantly (in separate formulations) wi
An adequate, well-controlled, double-blind, randomized study to evaluate the safety and efficacy of a dose of the netupitant/palonosetron fixed combination compared to standard therapy in pediatric cancer patients ages 0 to 17 years undergoing
In vivo drug interaction study to evaluate the duration of inhibitory effects of Akynzeo (netupitant and palonosetron) on CYP3A4 enzyme activity beyond 4 days after Akynzeo (netupitant and palonosetron) administration.
Conduct a study to evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response) of ledipasvir/sofosbuvir in pediatric subjects 3 to 17 years of age with chronic hepatitis C.
Enrollment for the study is currently ongoing. The Sponsor reports 78 subjects have been enrolled in Group 2 as of this reporting period.
Collect and analyze long-term safety data for subjects enrolled in the pediatric ledipasvir/sofosbuvir safety, pharmacokinetic and efficacy study. Data collected should include at least 3 years of follow-up in order to characterize the long-ter
Enrollment for the study is currently ongoing. The Sponsor reports 168subjects have been enrolled as of this reporting period.
Submit the ledipasvir 2 year rat carcinogenicity study
Submit the final report and datasets for the ongoing trial GS-US-337-0123, entitled “A Phase 2, Multicenter, Open-Label Study to Investigate the Safety and Efficacy of Sofosbuvir/Ledipasvir Fixed-Dose Combination + Ribavirin Administered in S
Submit the final report and datasets for the ongoing trial GS-US-337-0115, entitled “A Phase 3, Multicenter, Open-Label Study to Investigate the Safety and Efficacy of Sofosbuvir/Ledipasvir Fixed-Dose Combination for 12 Weeks in Subjects with
Submit an interim report from the ongoing trial GS-US-248-0122, entitled, “A Long Term Follow-up Registry for Subjects Who Achieve a Sustained Virologic Response to Treatment in Gilead-Sponsored Trials in Subjects with Chronic Hepatitis C Inf
Deferred pediatric study under PREA: Conduct a multicenter clinical evaluation of safety and efficacy in pediatric patients ages 9-17 years of age of Lumason as a contrast agent in pediatric echocardiography. Evaluate the efficacy of Lumason co
Lumason® (Sulfur Hexafluoride Lipid-Type A Microspheres)
Complete a pharmacokinetic trial to determine an appropriate dose of osimertinib in patients with mild to moderate hepatic impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics in Patients with Impaired Hepati
A randomized, double-blind, 26-week, active-controlled clinical trial comparing Foradil Aerolizer (formoterol fumarate inhalation powder) and fluticasone propionate with fluticasone propionate to evaluate the risk of serious asthma outcomes (ho
Product withdrawn from US market. Study terminated. Revised milestone letter issued 12/11/15. Final Report Submission September 2016.
Conduct an adequate leachable safety assessment for the [...] rubber stopper used in your container closure system. This assessment must include leachable data from long-term stability studies testing at least three batches (taking into cons id
Study 1: A Phase 2, Open-Label, Multiple Dose Study to Evaluate the Pharmacodynamic Effects, Safety, and Tolerability of Veltassa (Patiromer Sorbitex Calcium) for Oral Suspension in Children and Adolescents 2 to 18 Years of Age with Hyperkalemi
Study 2: A Phase 2, Open-Label, Multiple Dose Study to Evaluate the Pharmacodynamic Effects, Safety, and Tolerability of Veltassa (Patiromer Sorbitex Calcium) for Oral Suspension in Infants and Toddlers Under 2 Years of Age with Hyperkalemia
Submit integrated safety analyses and supporting data from an adequate number of clinical trial(s) to characterize the risk of cardiomyopathy and its sequelae in patients receiving trabectedin; to identify risk factors for development of these
Submit the final report of the completed clinical pharmacokinetic trial to determine the pharmacokinetics of Yondelis (trabectedin) in patients with hepatic impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetic
Deferred pediatric study under PREA for the treatment of schizophrenia in children aged 13 to 17 years. Conduct a Phase 3, Efficacy: multicenter, randomized, double-blind trial with two phases: Phase 1 - placebo- and activecontrolled, short-ter
Deferred pediatric study under PREA for the treatment of schizophrenia in adolescents aged 13 to17 years. Conduct a Phase 3, Safety: open-label, multicenter, long-term (2 years) study to obtain data on the safety of brexpiprazole in the relevan
A placebo-controlled, randomized withdrawal maintenance study of brexpiprazole in patients who require adjunctive treatment of major depressive disorder.
A placebo-controlled, randomized withdrawal maintenance study of brexpiprazole in patients with schizophrenia.
The merged protocol, Study 191622-137, was developed from the pediatric protocol Study 191622-137 (initial study) and Study 191622-138 (long-term extension study), i.e., the originally submitted Post Marketing Required studies per the OAB Appro
Study 191622-137 is a Multicenter, randomized, double-blind, parallel-group, multiple-dose study. The study is currently ongoing with 30 subjects enrolled. Allergan plans to enroll approximately 108 subjects in this study.
Conduct an assessment of neutralizing anti-drug antibody (ADA) response in serum samples of pediatric patients from Study PB-06-006 using a validated assay (developed under PMR 1895-2) that is capable of sensitively detecting ADA responses that
Evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of Elelyso (taliglucerase alfa) in at least 5 patients with body weight less than 15 kg; at least 5 patients with body weight 15-20 kg; and at least 5 patients with body weig
Conduct epidemiologic investigations to address whether the properties intended to deter misuse and abuse of Hysingla ER (hydrocodone bitartrate extended-release tablets) actually result in a significant and meaningful decrease in misuse and ab
Conduct a study to identify and quantify low molecular weight impurities in the [...]  polyethylene oxide (PEO) products used to manufacture Hysingla ER. Submit a toxicological risk assessment for the exposure to the impurities taking into cons
An observational study of pediatric patients from birth to 3 years of age who are administered iopromide for clinical evaluation, in order to assess their risk of developing hypothyroidism.
An observational study of pediatric patients from birth to 3 years of age who are administered iodixanol for clinical evaluation, in order to assess their risk of developing hypothyroidism.
An observational study of pediatric patients from birth to 3 years of age who are administered iodipamide meglumine for clinical evaluation, in order to assess their risk of developing hypothyroidism.
An observational study of pediatric patients from birth to 3 years of age who are administered ioxaglate meglumine; ioxaglate sodium for clinical evaluation, in order to assess their risk of developing hypothyroidism.
Pharmacokinetic, efficacy, and safety study or studies of Nucynta for the management of moderate to severe acute pain in pediatric patients ages birth to less than 17 years.
Per FDA letter dated 07/29/2016, this PMR has been fulfilled.
A randomized, double-blind, adequately controlled, multiple fixed doses, parallel group clinical study of Dysport(abobotulinumtoxinA) in botulinum toxin-naive children age 2-17 years with upper extremity spasticity. The minimum duration of the
A Pregnancy Pharmacovigilance Study to evaluate pregnancy outcomes and infant outcomes following exposure to Yervoy (ipilimumab). This study will include a mechanism to collect, classify, and analyze data on direct exposures (women exposed to i
Final protocol submission 18-DEC-15; agency comments received 08-FEB-16 teleconference (FDA and BMS) 12- FEB-16; missed milestone letter sent 07-MAR-16 Revised protocol sent 07-APR-16
Submit the final clinical report and datasets at the time of final analysis for overall survival (OS) of Trial CA184029, entitled “Adjuvant immunotherapy with anti-CTLA-4 monoclonal antibody (ipilimumab) versus placebo after complete resectio
Submit the final clinical report and datasets of Trial E1609, entitled “A Phase III Randomized Study of Adjuvant Ipilimumab Anti-CTLA4 Therapy Versus High Dose Interferon a-2b for Resected High Risk Melanoma” to inform any change to the rec
Conduct a long-term prospective observational study (product exposure registry), which includes a sufficient number of new Potiga-treated patients to assess baseline (i.e., prior to Potiga therapy) ophthalmologic test results and subsequent pot
Commitment to support the availability, through the use of clinical trial data, of an in-vitro diagnostic device that is essential to the safe and effective use of crizotinib for patients with ROS1 rearrangements in metastatic non-small cell lu
Complete a clinical trial to evaluate the effect of a strong CYP3A4 inhibitor on the pharmacokinetics of osimertinib. Conduct this trial in accordance with the FDA draft Guidance for Industry entitled “Drug Interaction Studies – Study Desig
Complete a clinical trial to evaluate the effect of a strong CYP3A4 inducer on the pharmacokinetics of osimertinib in accordance with the FDA draft Guidance for Industry entitled “Drug Interaction Studies – Study Design, Data Analysis, Impl
Complete a clinical trial to evaluate the effect of repeated doses of osimertinib on the pharmacokinetics of a probe substrate of CYP3A4 in accordance with the FDA draft Guidance for Industry entitled “Drug Interaction Studies – Study Desig
Complete a clinical trial to evaluate the effect of repeated doses of osimertinib on the pharmacokinetics of a probe substrate of breast cancer resistant protein (BCRP) in accordance with the FDA draft Guidance for Industry entitled “Drug Int
A juvenile rat toxicology study is required to identify the unexpected, serious risk of adverse effects of Xeomin (incobotulinumtoxinA) on postnatal growth and development. The study should utilize animals of an age range and stage(s) of develo
Per FDA letter dated 07/15/2016, this PMR has been fulfilled
Randomized, double-blind, adequate and well controlled, multiple fixed-dose, parallel group clinical trial of Xeomin (incobotulinumtoxinA) in botulinum toxinnaïve children age 2-17 years with upper extremity spasticity. The minimum duration of
A randomized, controlled, clinical trial to evaluate the safety of lesinurad 200mg on a background of concomitant xanthine oxidase inhibitor, with respect to renal function and renal adverse events, in gout patients who have not achieved target
Conduct a pediatric study under the Pediatric Research Equity Act (PREA) to evaluate the efficacy and safety, including sparse pharmacokinetic (PK) sampling, of Onzetra Xsail (sumatriptan) for the acute treatment of migraine in pediatric patien
Onzetra™ and Xsail™ (Sumatriptan Succinate)
Conduct a pediatric study under the Pediatric Research Equity Act (PREA) for the efficacy and safety of Onzetra Xsail (sumatriptan), including sparse pharmacokinetic sampling, for the acute treatment of migraine in pediatric patients ages 6 to
Genzyme commits to designing and implementing a registry of patients with Pompe disease being treated with alglucosidase alfa that will be established to obtain long-term clinical status information.  Information will be collected on patient de
Submit cumulative safety analyses annually, and for one year after the last patient has completed clinical trial treatment, to identify and characterize the risk of cardiomyopathy and subsequent sequelae, including safety evaluations adequate t
Using data from agreed upon studies of the component products, conduct and submit an assessment of safety, pharmacokinetics, and antiviral activity of ODEFSEY® (emtricitabine, rilpivirine, and tenofovir alafenamide) in pediatric patients 6 yea
Odefsey® (Emtricitabine, Rilpivirine, and Tenofovir Alafenamide)
An adequate randomized, active control, single-blind trial to evaluate renal dysfunction and laboratory abnormalities in adult patients, including elderly patients, patients with renal impairment, and patients with hepatic impairment taking Suc
Conduct and submit the results of a multicenter, randomized trial or trials establishing the superiority of nivolumab over standard therapy in adult patients with unresectable or metastatic melanoma who are refractory to ipilimumab or who have
Conduct an embryo-fetal development study in the rat model to assess the potential impact of polyethylene oxide (PEO) on development. The study must be designed to adequately qualify the safety of the low molecular weight PEO components (impuri
Conduct an embryo-fetal development study in the rabbit model to assess the potential impact of polyethylene oxide (PEO) on development. The study must be designed to adequately qualify the safety of the low molecular weight PEO components (imp
Conduct a pre- and post-natal development study in the rat model to assess the potential impact of polyethylene oxide (PEO) on development. The study must be designed to adequately qualify the safety of the low molecular weight PEO components i
Conduct a study to determine an appropriate dose and dosing interval for fexofenadine and pseudoephedrine in children, 6 to less than 12 years of age, who may benefit from the pseudoephedrine component of the drug product (i.e., not in otherwis
Allegra–D® Allergy and Congestion (Fexofenadine Hydrochloride and Pseudoephedrine Hydrochloride )
Conduct a study in children 6 to less than 12 years of age to evaluate the efficacy and safety of the pseudoephedrine component, in your combination product, for temporary relief of nasal congestion due to the common cold.
If the studies conducted in children 6 to less than 12 years of age demonstrate safety and efficacy for the intended claim, conduct a study to determine an appropriate dose and dosing interval for use in children, 2 to less than 6 years of age,
If the studies conducted in children 6 to less than 12 years of age demonstrate safety and efficacy for the intended claim, conduct a study to evaluate the safety of the pseudoephedrine component, in your combination product, for temporary reli
The study has not been initiated, but does not meet the criterion for delayed. Will begin in the future
Conduct a clinical trial to evaluate the incidence of intolerable grade 2 and grade 3-5 adverse reactions of an oral starting dose of lenvatinib 14 mg daily with everolimus 5 mg daily compared to the starting dose of lenvatinib 18 mg daily with
Conduct an open-label safety trial in at least 100 evaluable pediatric subjects with mild to moderate atopic dermatitis ages 3 months to < 2 years and at least 5% treatable percent body surface area (%BSA). Evaluate the pharmacokinetics of cris
Submit the progression-free survival (PFS) and overall survival (OS) analyses with datasets from clinical trial CO-338-014 entitled, “Phase 3 Study of Rucaparib as Switch Maintenance After Platinum in Relapsed High Grade Serous and Endometrio
Submit the progression-free survival (PFS) and overall survival (OS) analyses with datasets from clinical trial CO-338-043 entitled “A Phase 3 Multicenter, Randomized Study of Rucaparib Versus Chemotherapy in Patients With Relapsed, BRCA Muta
Conduct a dedicated PK trial in patients with moderate hepatic impairment to determine the appropriate starting dose in patients with moderate hepatic impairment.
Complete the ongoing drug-drug interaction trial CO-338-044 and submit the final study report.
Submit to FDA the appropriate analytical and clinical validation of a companion diagnostic test that will enable the evaluation of patients’ germline BRCA status.
Submit the final report of ongoing mass balance trial CO-338-045.
Conduct a human factors validation study to demonstrate that the user interface of the product can support safe and effective use for the intended users. The human factors validation study should be conducted in patients aged 6 years and older
Complete the ongoing clinical pharmacokinetic trial to determine an appropriate dose of Lonsurf (trifluridine and tipiracil) in patients with moderate to severe hepatic impairment in accordance with the FDA Guidance for Industry entitled “Pha
Complete the ongoing clinical pharmacokinetic trial to determine an appropriate dose of Lonsurf (trifluridine and tipiracil) in patients with severe renal impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics
A single-dose, open-label, randomized pharmacokinetic study of ADZENYS XR-ODT (amphetamine extended-release orally disintegrating tablets), in male and female children (4 to less than 6 years of age) with ADHD.
A randomized, double-blind, placebo-controlled, flexible-dose titration study of ADZENYS XR-ODT (amphetamine extended-release orally disintegrating tablets), in children ages 4 to 5 years diagnosed with ADHD.
A one year Pediatric Open-Label Safety Study of patients age 4 to 5 years (at the time of entry into PMR 3029-1 or PMR 3029-2, or at the time of enrollment if directly enrolled into PMR 3029-3) diagnosed with ADHD treated with ADZENYS XR-ODT (a
Conduct a study to characterize the safety of long-term exposure to Imbruvica based on data and pooled analyses from trials of patients with mantle cell lymphoma and chronic lymphocytic leukemia. Submit 3-year, 4-year, and 5-year safety follow-
Conduct an open-label, single- and multiple-dose, single-treatment, multi-center study to evaluate the pharmacokinetics and safety of XTAMPZA ER (oxycodone) extended-release capsules in patients 7-17 years.
Conduct an open-label, single- and multiple-dose, single-treatment, multi-center study to evaluate the pharmacokinetics and safety of XTAMPZA ER (oxycodone) extended-release capsules in patients 2-7 years.
In order to provide the baseline data to support the hypothesis-testing studies required under PMR 2966-4, conduct a descriptive study that analyzes data on the following: 1) utilization of XTAMPZA ER (oxycodone) extended release capsules and s
Conduct formal observational studies to assess whether the properties intended to deter misuse and abuse of XTAMPZA ER (oxycodone) extended release capsules actually result in a meaningful decrease in misuse and abuse, and their consequences, a
Conduct a chronic (6-month) repeat-dose general toxicology study in the rat model testing a mixture of beeswax, carnauba wax, and myristic acid that is representative of the drug product composition.
Submit integrated safety analyses from an adequate number of randomized controlled clinical trial(s) using Mekinist (trametinib) to identify and characterize the risk of retinal pigmented epithelial detachments (RPED), including safety evaluati
Submit cumulative safety analyses annually, and for one year after the last patient has completed clinical trial treatment, to identify and characterize the risk of new malignancies, including cutaneous squamous cell carcinoma, in all ongoing a
A clinical trial of patients with symptomatic neurogenic orthostatic hypotension to assess sustained effects of droxidopa therapy. The trial design consists of a 3-month, open-label droxidopa treatment period, followed by a 3-month, randomized,
Conduct a juvenile toxicity study in rats to support treatment of children and adolescents with AKOVAZ (ephedrine sulfate) injection by IV administration.
Conduct a single open-label study to evaluate the safety and pharmacodynamics/ pharmacokinetics of intravenous ephedrine dosed on a mg/kg basis with provisions for up-titration (dose or frequency) as clinically indicated in patents 12-16 years.
Conduct a fertility and early embryonic development toxicology study in the rat model for ephedrine sulfate.
Conduct an embryo-fetal developmental toxicology study using the rat model for ephedrine sulfate.
Conduct an embryo-fetal developmental toxicology study using the rabbit model for ephedrine sulfate.
Conduct a pre- and post-natal developmental toxicology study in the rat model for ephedrine sulfate.
Conduct an in vivo micronucleus genotoxicity assay with ephedrine sulfate.
Design and conduct a prospective trial and provide the full final report and data sets to evaluate dose reductions in patients who achieve a response or have stable disease in order to optimize the safety and efficacy of chronic administration
Submit the complete final report and data showing clinical efficacy and safety from trial GS-US-313-0124, a Phase 3, 2-arm, randomized, double-blind, placebo-controlled, parallel-group trial of idelalisib in combination with rituximab in subjec
Study terminated March 11, 2016. 295 subjects had been enrolled
Submit the complete final report and data showing clinical efficacy and safety from trial GS-US-313-0125, a Phase 3, 2-arm, randomized, double-blind, placebo controlled, parallel-group trial of idelalisib in combination with bendamustine plus r
Study terminated March 11, 2016. 475 subjects had been enrolled
Conduct a study to characterize the incidence, diagnosis, and effective treatment of Zydelig-related pneumonitis based on data and pooled analyses from randomized trials in iNHL and CLL (0115, 0119, 0124, and 0125).
Conduct a trial to provide evidence sufficient to characterize the long-term safety of Zydelig. Submit the complete final report and data showing long-term safety with 5 years of follow-up from trial 101-99 Phase 1/2 extension study of safety a
Conduct a trial to provide evidence sufficient to characterize the long-term safety of Zydelig when used in combination with other agents such as bendamustine (B) and rituximab (R). Submit the complete final report and data showing long-term sa
Study terminated March 11, 2016. 457 subjects had been enrolled
Conduct a trial to provide evidence sufficient to characterize the long-term safety of Zydelig when used in combination with an anti-CD20 drug regimen. Submit the complete final report and data from trial GS-US-312-0119, a Phase 3, randomized,
Conduct a trial to provide evidence sufficient to characterize the long-term safety of Zydelig when used in a combination therapy regimen. Submit the complete final report and data showing long-term safety with 5 years of follow-up from trial G
Deferred pediatric study under PREA: Conduct a pharmacokinetic and safety study of an age-appropriate formulation of oxycodone hydrochloride/naloxone hydrochloride extended-release tablets in patients from ages 7 to less than 17 years with pain
Conduct epidemiologic investigations to address whether the properties intended to deter misuse and abuse of TARGINIQ ER (oxycodone hydrochloride and naloxone hydrochloride extended-release tablets) actually result in a significant and meaningf
Conduct a combination in vivo micronucleus and comet assay for [...] The comet assay portion of the study should include assessment of both stomach and liver tissue and include doses of the drug substance that would be obtained at the maximum r
Conduct a chronic (9-month) repeat-dose general toxicology study in the dog model testing a mixture of beeswax, carnauba wax, and myristic acid that is representative of the drug product composition.
Conduct a fertility and early embryonic development study in the rat model testing a mixture of beeswax, carnauba wax, and myristic acid that is representative of the drug product composition.
Conduct an embryo-fetal development study in the rat model testing a mixture of beeswax, carnauba wax, and myristic acid that is representative of the drug product composition.
Conduct an embryo-fetal development study in the rabbit model testing a mixture of beeswax, carnauba wax, and myristic acid that is representative of the drug product composition.
Conduct a pre- and post-natal development study in the rat model testing a mixture of beeswax, carnauba wax, and myristic acid that is representative of the drug product composition.
Conduct a carcinogenicity assessment in the rat model testing a mixture of beeswax, carnauba wax, and myristic acid that is representative of the drug product composition.
Conduct a carcinogenicity assessment in the mouse model testing the mixture of beeswax, carnauba wax, and myristic acid that is representative of the drug product composition.
Complete a detailed analysis of the beeswax employed in your drug product for potential residual levels of environmental and apicultural sources of contaminants, based on a thorough review of the apicultural practices across the globe and known
Conduct a study to characterize the levels of [l...] in the final drug product formulation and propose a release specification to adequately control [...] in the drug product.
A long-term, open-label, safety study of adjunctive therapy in patients from 1 month to less than 12 years of age with epilepsy. The purpose of this study is to evaluate the long-term safety of FYCOMPA (perampanel) as adjunctive therapy in the
Submit the final report and efficacy datasets for Keynote-024, entitled “A Randomized, Open-Label, Phase 3 Trial of MK-3475 versus Platinum-Based Chemotherapy in 1L Subjects with PD-L1 Strong, Metastatic Non-Small Cell Lung Cancer” to updat
Submit the final report and efficacy datasets for Keynote-042, entitled: “A Randomized, Open Label, Phase III Study of Overall Survival Comparing Pembrolizumab (MK-3475) versus Platinum Based Chemotherapy in Treatment Naïve Subjects with PD-
In order to provide the baseline data to support the hypothesis-testing studies required under PMR 3148-2, conduct a descriptive study that analyzes data on the following: (1) utilization of ARYMO ER and selected comparators. Reports should inc
Submit integrated safety analyses of cardiac valve abnormalities based on centralized, blinded, independent review assessment of all echocardiograms from an adequate number of randomized controlled clinical trials that use Tafinlar (dabrafenib)
Submit a final report of follow-up safety data of Xgeva (denosumab) in patients with giant cell tumor of bone enrolled in the ongoing single arm trial through November 2012 for a minimum of five years or until death or lost to follow-up, whiche
Submit the final report including primary datasets, derived datasets, and analysis programs used to generate the safety and efficacy results for the ongoing single arm multicenter trial of denosumab in patients with giant cell tumor of bone. In
A post-marketing, observational epidemiologic study comparing oral Relistor (methylnaltrexone bromide) to other treatments of opioid induced constipation in patients with chronic non-cancer pain. The study’s primary outcome is a composite of
A two-year carcinogenicity study of intravenously administered eteplirsen in rat.
A 26-week carcinogenicity study of eteplirsen, administered by a clinically relevant route, in an appropriate transgenic mouse model.
Pharmacokinetic/safety study of tavaborole topical solution, 5% in 40 pediatric subjects age 12 to 17 years and 11 months with onychomycosis of the toenails. Pharmacokinetic assessments will be done in at least 16 evaluable subjects under maxim
Conduct a 52-week, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of saxagliptin vs. placebo, both as add-on therapy to metformin in pediatric patients with inadequate glycemic control on metformin alone.
Deferral Extension Requested 04/15/2016. Denied per FDA letter dated 04/25/2016.
Kombiglyze® XR (Saxagliptin and Metformin Hydrochloride)
A postmarketing observational cohort study comparing TARGINIQ ER (oxycodone hydrochloride/naloxone hydrochloride) to other drugs approved for the management of pain severe enough to require daily, around-the-clock, longterm opioid treatment and
Based on review of your April 27, 2015 submission, which you submitted with the intention of fulfilling PMR 2762-4, FDA has determined that have good cause for your failure to comply with the final protocol submission milestone of the original
Submit the final report and datasets for HPC3017 (OPTIMIST 1).
Per FDA letter dated 05/20/2016, this PMR has been fulfilled.
Deferred pediatric study under PREA for the use of Atripla alone as a complete regimen or in combination with other antiretroviral agents for the treatment of HIV-1 infection in pediatric subjects from 6 to less than 12 years of age.
Per FDA letter dated 07/13/2016, this PMR has been released.
Atripla® (Efavirenz, Emtricitabine, and Tenofovir Disoproxil Fumarate)
Submit the final report and datasets for HPC3018 (OPTIMIST-2).
To develop a presentation that can be used to directly and accurately administer filgrastim-sndz to pediatric patients who weigh less than 36 kg requiring doses that are less than 0.3 mL (180 mcg), and conduct any necessary human factors studie
Original Final Report Due Date: 09/06/2016; Deferral Extension granted per FDA letter dated 12/09/2016.
Conduct an efficacy and safety study evaluating Repatha (evolocumab) in patients with heterozygous familial hypercholesterolemia (HeFH) ages 10 years to less than 18 years. The study will be a randomized, 6-month, double-blind, placebocontrolle
Conduct a prospective observational study of pregnant women exposed to Repatha (evolocumab) to evaluate fetal, infant, and childhood outcomes of pregnant women exposed to evolocumab and their live born offspring through the first 5 years of lif
Conduct a large, randomized, controlled, long-term trial in which the incidence and severity of new-onset diabetes mellitus, injection site reactions, hypersensitivity, immunogenicity, and adverse events potentially related to demyelination wit
Conduct a randomized, controlled, long-term trial that prospectively evaluates changes in neurocognitive function with Repatha (evolocumab) treatment. The trial must be adequately powered to exclude a clinically meaningful adverse effect.
Conduct formal observational studies to assess whether the properties intended to deter misuse and abuse of ARYMO ER actually result in a meaningful decrease in misuse and abuse, and their related clinical outcomes, addiction, overdose, and dea
Provide a detailed and thoughtful analysis of the risk factors associated with malignant transformation of GCTB and development of new sarcoma and the lifetime and annual incidences of these events in denosumab naïve patients. For this analysi
Conduct a fertility and early embryonic development toxicology study in the rat model for phenylephrine hydrochloride completed via modern reproductive and developmental toxicology studies, as outlined in the guidance for industry, Detection of
Conduct an embryo-fetal developmental toxicology study using the rat model for phenylephrine hydrochloride completed via modern reproductive and developmental toxicology studies, as outlined in the guidance for industry, Detection of Toxicity t
Conduct an embryo-fetal developmental toxicology study using the rabbit model for phenylephrine hydrochloride completed via modern reproductive and developmental toxicology studies, as outlined in the guidance to industry, Detection of Toxicity
Conduct a peri- and post-natal developmental toxicology study in the rat model for phenylephrine hydrochloride completed via modern reproductive and developmental toxicology studies, as outlined in the guidance for industry, Detection of Toxici
A postmarketing study that will allow you to test for and control the levels of the following [...] impurities in your product: [...] that are present during the manufacturing process and have the potential to contaminate your product. As part
The final study reports for the remaining products subject to the postmarketing requirement have not been submitted because the FDA was engaged in discussion with the applicant regarding the study design at the time the final study reports subm
Resectisol® (Mannitol Irrigation) in PIC™ (Plastic Irrigation Container)
Isolyte® H in EXCEL® Plastic Container (Dextrose and Multiple Electrolytes)
Isolyte® R in EXCEL® Plastic Container (Dextrose and Multiple Electrolytes)
Isolyte® E in EXCEL® Pastic Container (Dextrose and Multiple Electrolytes)
Isolyte® P in EXCEL® Pastic Container (Dextrose and Multiple Electrolytes)
Dextrose in Ringer's Injection in EXCEL® Plastic Container
Calcium Choride and Potassium Chloride in Dextrose
Conduct a juvenile rat toxicology study to identify the unexpected serious risk of adverse effects of zolmitriptan on postnatal growth and development. The study should utilize animals of an age range and stage(s) of development that are compar
Submit an enhanced pre and post-natal development study (segment III) in rats to demonstrate the safety of lansoprazole on developing bone. The studies must include at least 3 dose levels of lansoprazole and a control group and should include t
Submit an enhanced pre and post-natal development study (segment III) in rats to demonstrate the safety of lansoprazole on developing bone. The studies must include at least 3 dose levels of lansoprazole and a control group and should include t
Submit an enhanced pre and post-natal development study (segment III) in rats to demonstrate the safety of pantoprazole sodium on developing bone. The studies must include at least 3 dose levels of pantoprazole sodium and a control group and sh
Conduct a long-term safety trial, including assessment of calcium metabolism, of Vectical® (calcitriol) Ointment in 100 evaluable pediatric subjects with plaque psoriasis aged 2 to 16 years and 11 months. Pharmacokinetic/Pharmacodynamic (calci
Conduct an open-label study to assess safety, pharmacokinetics, and treatment effect of ACZONE (dapsone) Gel, 7.5% in 100 pediatric subjects aged 9 years to 11 years 11 months with acne vulgaris. Pharmacokinetic assessments will be done in at l
A prospective, randomized, controlled, double-blind, parallel-group study to compare the safety and efficacy of SMOFLIPID (lipid injectable emulsion) to standard- of- care soybean oil based lipid emulsion in hospitalized neonates including low
SMOFlipid™ (Soybean Oil, Medium Chain Triglycerides, Olive Oil, and Fish Oil)
Conduct a long-term, postmarketing, observational study to assess the long-term safety of STELARA (ustekinumab) versus other therapies used in the treatment of adults with moderate to severe Crohn’s disease. The study’s primary outcome is m
Conduct a dose-ranging trial to determine the pharmacokinetics/pharmacodynamics, safety, and tolerability of STELARA (ustekinumab) induction dosing in pediatric patients 2 to 17 years of age with moderately to severely active Crohn’s disease
Conduct a randomized, controlled, blinded, multicenter trial to evaluate the safety and efficacy of STELARA (ustekinumab) in pediatric patients 2 to 17 years of age with moderately to severely active Crohn’s disease despite conventional thera
Conduct a clinical trial to assess whether STELARA (ustekinumab) alters the metabolism or pharmacokinetics of cytochrome P450 (CYP) substrates in Crohn’s disease (CD) patients treated with ustekinumab (e.g., using a cocktail of relevant CYP p
[...] Identify and quantitate the impurities. Submit a toxicological risk assessment for the exposure to the impurities taking into consideration the maximum theoretical daily dose of ARYMO ER.
Conduct an embryo-fetal development study in the rat model to assess the potential impact [...] on development. The study must be designed to adequately qualify the safety [...] when the product is consumed up to the MTDD of ARYMO ER.
Conduct an embryo-fetal development study in the rabbit model to assess the potential impact [...] on development. The study must be designed to adequately qualify the safety [...] when the product is consumed up to the MTDD of ARYMO ER.
Conduct a pre- and post-natal development study in the rat model to assess the potential impact [...] on development. The study must be designed to adequately qualify the safety [...] when the product is consumed up to the MTDD of ARYMO ER.
There is some preliminary evidence of [...] which could potentially alter the abuse-deterrent properties of the drug product. [...]
Conduct a controlled efficacy and pharmacokinetics (PK) study in children ages > or equal to 6 years to 11 years with migraine that includes sparse PK samples throughout the efficacy study. Conduct a long-term open-label safety study in pediatr
A study to evaluate: 1. patient immune responses, including IgM and IgG isotypes, to eteplirsen, its induced dystrophin protein, and full length dystrophin; 2. the impact of immune responses on product PK and clinical efficacy and safety. The a
Conduct a 2-year controlled trial in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 or 53 skipping with a phosphorodiamidate morpholino oligomer (PMO) designed to bind to a regulatory site governing splicing
Continue to evaluate the long-term efficacy and safety of XURIDEN (uridine triacetate) in patients currently enrolled in Protocol 401-13-001 every 6 months for a total duration of 2 years in an extension trial. The extension trial should collec
Conduct a 12-week, randomized, double-blind, parallel-group, placebocontrolled, efficacy and safety study of tiotropium delivered via the Respimat device in children 6-11 years of age with asthma. The study should evaluate at least two doses of
The final report was submitted to FDA on 08/15/2016.
Conduct a 48-week, randomized, double-blind, parallel-group, placebocontrolled, efficacy and safety study of tiotropium delivered via the Respimat device in children 6 to 11 years of age with asthma. The study should evaluate at least two doses
Conduct a study in adult healthy volunteers to assess the pharmacokinetics and bioavailability of multiple age-appropriate tofacitinib XR formulations compared to the tofacitinib IR formulation.
Conduct a pharmacokinetic study in adult healthy volunteers comparing multiple tofacitinib XR formulations to the tofacitinib IR formulations to select a suitable age-appropriate tofacitinib XR formulation that meets the target pharmacokinetic
Conduct a pharmacokinetic study in adult healthy volunteers to establish the equivalence of AUC and similarity in other pharmacokinetic parameters between the proposed commercial age-appropriate tofacitinib XR formulation and tofacitinib IR for
A prospective descriptive observational cohort study of insertion-, localization-, and removal-related serious adverse events and their sequelae associated with PROBUPHINE (buprenorphine hydrochloride) use.
Clinical trial to assess the risk of QT prolongation with subdermal PROBUPHINE (buprenorphine hydrochloride).
Clinical trial to evaluate the effect of scarring or inflammation related to a prior implant on the safety of re-implantation/reinsertion, the potential for migration of implants, and the bioavailability of PROBUPHINE (buprenorphine hydrochlori
Clinical trial to evaluate the safety, feasibility, and pharmacokinetics of PROBUPHINE (buprenorphine hydrochloride) implantation at alternate body sites. The trial should also evaluate the safety of other methods of inserting PROBUPHINE (bupre
Conduct a pediatric trial to evaluate the safety and antiviral activity of COMPLERA(emtricitabine/rilpivirine/tenofovir disoproxil fumarate) FDC tablets in HIV infected pediatric subjects 12 to 18 years old. Safety and antiviral activity (effic
Complera® (Emtricitabine, Rilpivirine, and Tenofovir Disoproxil Fumarate)
Conduct a pediatric trial to evaluate the safety and antiviral activity of COMPLERA (emtricitabine/rilpivirine/tenofovir disoproxil fumarate) FDC tablets in HIV infected pediatric subjects 6 years to less than 12 years old. Safety and antiviral
Conduct a trial to investigate the effects on mortality of multiple doses of sildenafil in adults with pulmonary arterial hypertension.
Conduct a single-dose pharmacokinetic trial in subjects with mild, moderate, and severe hepatic impairment. Include overweight and obese subjects in the trial population.
Conduct a single-dose pharmacokinetic trial in subjects with mild, moderate, and severe renal impairment. Include overweight and obese subjects in the trial population.
Conduct a drug-drug interaction clinical trial with organic cation transporter 2 (OCT2) substrate, such as metformin, to evaluate the in vivo potential of Contrave constituents (bupropion and naltrexone) to inhibit OCT2. The trial should test t
Randomized controlled trial to evaluate the safety and efficacy of SMOFLIPID (lipid injectable emulsion) administered for at least 90 days in pediatric patients, compared to standard of care soybean oil based lipid emulsion administered for the
Test the three registration stability batches for the individual component phytosterol content in SMOFLIPID (lipid injectable emulsion) using the validated analytical methods.
Test for the individual component phytosterol content in all batches of SMOFLIPID (lipid injectable emulsion) manufactured over a three year period, using the validated analytical method. Based on these test results, establish safety limits for
Randomized clinical trial in hospitalized adult patients receiving either SMOFLIPID (lipid injectable emulsion) or other standard-of-care IV lipid emulsions to evaluate clinical safety outcomes of sepsis and mortality. The trial will also evalu
Randomized controlled trial in pediatric patients, including neonates, comparing a phytosterol-depleted formulation of SMOFLIPID (lipid injectable emulsion) and another standard-of-care lipid emulsion (100 % soybean oil product) to evaluate the
Conduct a study to assess the pharmacokinetics of each drug component of hydrocodone and guaifenesin in children ages 6 to 17 years with symptoms of the common cold.
Protocol submitted on March 31, 2015.The study has not begun but does not fit criteria for delay.
Obredon® (Hydrocodone Bitartrate and Guaifenesin)
Conduct a study to assess the safety of hydrocodone and guaifenesin in children ages 6 to 17 years with symptoms of the common cold. The dose used in this study will be based upon the results of the pharmacokinetic study in children ages 6 to 1
A prospective, registry-based observational exposure cohort study conducted in the United States, that compares the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to Lemtrada (alemtuzumab) during pregnancy to unex
A pre- and postnatal development (including maternal function) study of Lemtrada (alemtuzumab) in rHu CD52 mouse.
A prospective observational registry study in adult patients with relapsing multiple sclerosis, with the primary objective of determining the necessary duration of monitoring following treatment with Lemtrada (alemtuzumab) for multiple sclerosi
A prospective study in adult patients with relapsing multiple sclerosis to assess patient safety during and after Lemtrada (alemtuzumab) infusion in multiple sclerosis patients. Measurements of interest include: 1) Duration of infusion; 2) Vita
Submit the final overall survival (OS) data from Trial CA209037, a randomized, open-label, Phase 3 Trial of Nivolumab Versus Investigator’s Choice in Advanced (Unresectable or Metastatic) Melanoma Patients Progressing Post Anti-CTLA-4 Therapy
Conduct epidemiologic investigations to address whether the properties intended to deter misuse and abuse of MORPHABOND (morphine sulfate extended release tablets) actually result in a significant and meaningful decrease in misuse and abuse, an
Conduct a 9-month repeat-dose oral toxicology study in the nonrodent model characterizing the toxicological potential of [...]
Conduct a 6-month repeat-dose oral toxicology study in the rodent model characterizing the toxicological potential of [...]
Conduct a 2-year rodent oral carcinogenicity assessment of [...]
Randomized clinical trial comparing SMOFLIPID (lipid injectable emulsion) to another standard-of-care IV lipid emulsion, evaluating long-term risk of developing essential fatty acid deficiency (EFAD) and parenteral nutrition associated liver di
An open-label trial of the safety and treatment effects of Sorilux Foam, 0.005% in 75 evaluable pediatric subjects with plaque psoriasis of the scalp and body age 2 years to 11 years and 11 months. Pharmacokinetic/Pharmacodynamic parameters wil
PMR newly issued in FDA letter dated 11/25/2015 to replace 1944-2.
An enhanced pharmacovigilance study of ketoacidosis in patients treated with dapagliflozin. The study will include reports of ketoacidosis or diabetic ketoacidosis for a period of 5 years, and will include assessment and analysis of spontaneous
An enhanced pharmacovigilance study of ketoacidosis in patients treated with canagliflozin. The study will include reports of ketoacidosis or diabetic ketoacidosis for a period of 5 years, and will include assessment and analysis of spontaneous
An enhanced pharmacovigilance study of ketoacidosis in patients treated with empagliflozin. The study will include reports of ketoacidosis or diabetic ketoacidosis for a period of 5 years, and will include assessment and analysis of spontaneous
Glyxambi (Empagliflozin and Linagliptin) Tablets
Conduct a Pharmacokinetic study in Attention Deficit Hyperactivity Disorder (ADHD) patients aged 4 to 5 years old.
Conduct a 6-week, double-blind, placebo-controlled, randomized, parallel-group safety and efficacy study in children with Attention Deficit Hyperactivity Disorder (ADHD) 4 to 5 years of age using methylphenidate ER formulation (QuilliChew ER)
Conduct a 6-month, open-label extension study to obtain additional information on safety and tolerability of methylphenidate ER formulation (QuilliChew ER) in children 4 to 5 years of age with Attention Deficit Hyperactivity Disorder (ADHD)
Develop a presentation that can be used to accurately administer etanercept-szzs to pediatric patients who weigh less than 63 kg.
Develop and validate an assay that is sufficiently sensitive, selective, and specific to reliably detect product specific antibodies of the IgE isotype.
Use the anti-reslizumab IgE assay developed under 3053-1 to test serum samples from patients who had treatment associated anaphylaxis.
Submit a qualification report demonstrating the suitability of the commercial antialpha gal ELISA from [...] that was used to analyze the sera of the four treatment-related anaphylaxis patients.
Conduct a phase 4 observational study to evaluate the efficacy and safety of Neupogen®(filgrastim) in the setting of Hematopoietic syndrome (HS) following acute radiation exposure.
A prospective, long-term, observational study in a routine clinical setting of patients aged 3 weeks or older with bile acid synthesis disorders due to single enzyme defects and patients with peroxisomal disorders, including Zellweger spectrum
The final protocol has not been submitted and final protocol milestone date has been missed.
Conduct a prospective observational study of patients enrolled in the Zinbryta (daclizumab) Risk Evaluation and Mitigation Strategies (REMS) registry, with the primary objective of determining the incidence rates of drug-induced liver injury, s
Conduct a prospective observational study of patients enrolled in the Zinbryt (daclizumab) REMS registry, with the primary objective of determining the incidence and mortality rates of breast cancer. All patients enrolled in the registry should
Conduct a nested case-control study among patients enrolled in the Zinbryta (daclizumab) REMS registry, with the primary objective of determining which clinical attributes are risk factors or protective factors for developing liver disorders an
Conduct prospective pregnancy exposure registry cohort analyses in the United States that compare the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to Zinbryta (daclizumab) during pregnancy with two unexposed con
Develop and validate an assay with improved sensitivity for the detection of neutralizing antibodies against daclizumab in the presence of daclizumab levels that are expected in samples collected from patients on treatment.
A randomized, placebo-controlled clinical trial to evaluate the safety, efficacy and steady-state pharmacokinetics of OCALIVA (obeticholic acid) in patients with primary biliary cholangitis (PBC) with Child-Pugh Classes B and C hepatic impairme
Establish the optimal and safe dose of belinostat in combination with the cyclophosphamide/vincristine/doxorubicin/prednisone (CHOP) regimen. Perform a Phase 1 dose finding trial of belinostat plus CHOP for the treatment of patients with periph
Characterize the comparative efficacy and safety of belinostat when used in combination with a treatment regimen such as CHOP, versus the combination of pralatrexate plus CHOP, versus CHOP alone for the initial therapy of patients with PTCL. Pe
Final Protocol Submission was due December 2015. No SPA agreement has been made.
Conduct an in vitro study to determine the exact contributions of UGT1A1, CYP3A4, CYP2C9, and CYP2A6 in the biotransformation of belinostat to evaluate the potential for higher drug exposure or metabolism to a potentially more toxic metabolite.
Characterize the mass balance information for Beleodaq. Submit the final clinical trial report for the ongoing human mass balance trial (Protocol SPI-BEL-12-103 designed to evaluate the excretion route of belinostat in humans. Excretion alterat
Characterize the PK of belinostat in the presence of strong UGT1A1 inhibitors. Conduct a clinical trial evaluating the influence of strong UGT1A1 inhibitors on the pharmacokinetics of belinostat in patients with cancer. Submit a complete final
The clinical trial completion date milestones have been missed and the final reports have not been submitted
Evaluate the safety and pharmacokinetics of belinostat in patients with wild-type, heterozygous, and homozygous UGT1A1*28 genotypes. The evaluations should be conducted for sufficient duration and in a sufficient number of subjects in order to
Characterize the PK and safety of belinostat in the presence of hepatic impairment. Submit the final clinical trial report for the ongoing hepatic impairment trial (Protocol CTEP #8846) that is designed to evaluate the influence of hepatic impa
Characterize the PK and safety of belinostat in the presence of renal impairment. Conduct a clinical trial in patients with varying degrees of renal impairment to evaluate the pharmacokinetic and safety of belinostat patients with impaired rena
Final Report was due March 2016. The study was initiated April 2016.
Submit the final report and datasets for clinical trial entitled “A Phase III, Open-Label, Multicenter, Randomized Study to Investigate the Efficacy and Safety of Atezolizumab (Anti-PD-L1 Antibody) Compared with Docetaxel in Patients with Non
Establish reliability requirements for the combination product EVZIO (naloxone hydrochloride injection) and complete testing that verifies combination product reliability.
Conduct case study analysis of reports of failure of the combination product EVZIO (naloxone hydrochloride injection) to activate, or failure of the combination product to deliver the full-labeled dose. Perform detailed analyses of reported dev
Conduct a randomized, double-blind, placebo-controlled trial of safety, efficacy, and pharmacokinetics of Zinplava (bezlotoxumab) in pediatric patients from 1 to less than 18 years of age receiving antibacterial therapy for C. difficile infecti
Conduct a study to evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response) of sofosbuvir and velpatasvir in pediatric subjects 12 through less than 18 years of age with chronic hepatitis C virus infecti
Conduct a study to evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response) of sofosbuvir and velpatasvir in pediatric subjects 3 through less than 12 years of age with chronic hepatitis C virus infectio
Conduct a drug interaction trial to evaluate the interaction between sofosbuvir and velpatasvir and atorvastatin.
Submit the final clinical report and datasets for the ongoing trial GS-US-342-1202 (ASTRAL-5), titled “A Phase 3, Open-label Study to Investigate the Efficacy and Safety of Sofosbuvir/GS-5816 Fixed Dose Combination for 12 weeks in Subjects wi
Conduct a trial in hepatitis C virus genotype 3 infected subjects with cirrhosis treated with sofosbuvir and velpatasvir to determine if the addition of ribavirin improves the efficacy (i.e., sustained virologic response rate) and reduces the r
Collect, analyze, and submit data from the HCV infected subjects with decompensated Child-Pugh Turcotte (CPT) C cirrhosis treated with sofosbuvir/velpatasvir regimen to obtain safety data in a broader decompensated cirrhosis population (genotyp
Collect, analyze, and submit data on subjects with cirrhosis including decompensated cirrhosis who achieve sustained virologic response following treatment with a sofosbuvir/velpatasvir-based regimen to evaluate durability of virologic response
Conduct site-directed mutant phenotypic analyses of sofosbuvir against an HCV genotype 3 replicon with the following substitutions: NS5B_L314F, NS5B_L314I, and NS5B_L314P.
Conduct a study to evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response) of daclatasvir in combination with other direct acting antivirals in pediatric subjects 3 through 17 years of age with chronic
Per FDA letter dated 08/08/2016, this PMR has been released.
Conduct an observational study to characterize the long-term (> 1 year) persistence of treatment-emergent daclatasvir resistance-associated substitutions in hepatitis C virus genotype 3 infected subjects who failed treatment with daclatasvir-co
Evaluate the potential mechanism of both pharmacodynamic and pharmacokinetic interactions between amiodarone and HCV direct acting antivirals, including daclatasvir, using a multielectrode array electrophysiology study in human stem-cell derive
Evaluate the effect of individual HCV direct acting antivirals including daclatasvir on the plasma protein binding of amiodarone using the TRANSIL® high sensitivity binding assay to help elucidate the potential mechanism of an interaction betw
Conduct a trial in hepatitis C virus genotype 3 infected subjects with cirrhosis treated with daclatasvir plus sofosbuvir to determine if a longer duration of treatment or the addition of ribavirin reduces the rate of virologic failure and the
Conduct a dose-finding study (Phase 2) and an efficacy and safety study (Phase 3) evaluating alirocumab in patients with heterozygous familial hypercholesterolemia (HeFH) ages 10 years to less than 18 years. If children younger than age 10 are
Delayed in meeting the due date for the Final Protocol Submission (Phase 2).
Conduct a prospective observational study of pregnant women exposed to Praluent to evaluate fetal, infant, and childhood outcomes of pregnant women exposed to Praluent and their live born offspring through the first 5 years of life to estimate
Conduct a large, randomized, controlled, long-term trial in which the incidence and severity of new- onset diabetes mellitus, injection site reactions, hypersensitivity, immunogenicity, and adverse events potentially related to demyelination wi
Conduct a randomized, controlled, long-term trial that prospectively evaluates changes in neurocognitive function with alirocumab treatment. The trial must be adequately powered to exclude a clinically meaningful adverse effect.
To develop an algorithm for decision-making in the presence of loss of efficacy due to antibody response. This should include an examination of the binding of alirocumab-specific neutralizing antibodies to the LDL receptor in patients in whom t
Conduct an epidemiologic study using claims or electronic health records data to evaluate the incidence of angioedema in Black patients treated with Entresto compared to a control drug. A target sample size, supported by sample size calculation
Entresto™ (Sacubutril and Valsartan) and Enalapril
A multicenter, randomized, double-blind, active-controlled trial to evaluate the effects of Entresto compared to valsartan on cognitive function as assessed by comprehensive neurocognitive battery and PET imaging in patients with chronic heart
A randomized, placebo-controlled trial to evaluate the safety and efficacy of OCALIVA (obeticholic acid) used as monotherapy in patients with primary biliary cholangitis (PBC) who are intolerant of or non-responsive to ursodeoxycholic acid (UDC
A randomized, double-blind, placebo-controlled trial to verify and describe that OCALIVA (obeticholic acid) induced reductions in alkaline phosphatase and/or total bilirubin are associated with improvements in the composite clinical endpoint of
Develop a formulation that would allow once daily dosing of OCALIVA (obeticholic acid) for patients with hepatic impairment. Conduct a study in healthy subjects to characterize the bioavailability of the new formulation relative to an approved
Conduct a pharmacokinetic and safety study of an age-appropriate formulation of VANTRELA ER in patients from ages seven to less than 17 years with pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which a
Vantrela ER (hydrocodone bitartrate) tablets
In order to provide the baseline data to support the hypothesis-testing studies required under PMR 2981-3, conduct a descriptive study that analyzes data on the following: 1) Utilization of VANTRELA ER and selected comparators. Reports should i
Conduct a multiple-dose pharmacokinetic (PK) and tolerability study to explore the range of tolerated doses of Fycompa in patients from 2 to less than 12 years old with epilepsy. A sufficient proportion of subjects must be on background therapy
The PK and Tolerability study component of PMR 2922-1 is being conducted via PMR 1932-2 and has been initiated. The Efficacy and Safety study component of PMR 2922-1 has not yet been initiated.
An efficacy, safety and dose-finding study of Qbrelis in hypertensive pediatric patients two years to less than six years of age
A postmarketing, prospective, observational, cohort study of vedolizumab versus other agents for inflammatory bowel disease. The study's primary outcome is serious infections. Secondary outcomes include, but are not limited to, progressive mult
Submit data from the randomized withdrawal period (Epoch 3) of the ongoing phase 3 study, CACZ885N2301 (N2301). Include an assessment of the maintenance of efficacy of canakinumab at a reduced dosing frequency in the study report.
Conduct an open-label, dose-finding, pharmacokinetics, safety and tolerability study of Orbactiv (oritavancin diphosphate) single dose infusion in pediatric subjects less than 18 years of age with suspected or confirmed bacterial infections.
Conduct a multicenter, evaluator-blind, randomized study to evaluate the safety and tolerability of single-dose IV Orbactiv (oritavancin diphosphate) versus  vancomycin for the treatment of pediatric subjects less than 18 years of age with acut
Final protocol submission date is September, 2017
Conduct a US surveillance study over a five-year period from the date of marketing Orbactiv (oritavancin diphosphate) to determine if resistance to oritavancin has developed in those organisms specific to the indication in the label for ABSSSI.
A randomized, double blind, multicenter, comparative study to establish the safety and tolerability profile of ceftolozane/tazobactam compared to that of meropenem in hospitalized children from birth to <18 years with cIAI. The dose for this st
Establish the efficacy and safety of APTIOM when used as monotherapy in the treatment of partial-onset seizures in patients 1 month to <18 years of age. This PMR may be fulfilled by a pharmacokinetic/pharmacodynamic analysis of data collected a
An open-label, long-term safety study to evaluate the long-term safety of APTIOM when used as monotherapy for partial-onset seizures in patients 1 month to <18 years of age.
Conduct formal observational studies to assess whether the properties intended to deter misuse and abuse of VANTRELA ER actually result in a meaningful decrease in misuse and abuse, and their consequences, addiction overdose, and death, in post
A multiple ascending dose thorough QT (tQT) clinical trial in healthy adult volunteers designed to determine the maximum tolerated dose of hydrocodone bitartrate without co-administration of naltrexone and characterize the effect of VANTRELA ER
Submit the complete final report and data from a randomized, Phase 3 trial, comparing ibrutinib in combination with bendamustine and rituximab or rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone versus bendamustine and ritu
Determine the appropriate Trulance (plecanatide) treatment dose for pediatric patients with chronic idiopathic constipation (CIC) who are 12 years to less than 18 years of age by assessing the safety and efficacy of once daily oral plecanatide
Determine the appropriate Trulance (plecanatide) treatment dose for pediatric patients with chronic idiopathic constipation (CIC) who are 6 years to less than 12 years of age by assessing the safety and efficacy of once daily oral plecanatide i
Confirm the efficacy and safety of Trulance (plecanatide) in pediatric patients with chronic idiopathic constipation (CIC) who are 6 years to less than 18 years of age by performing a randomized, double-blind, placebo-controlled, parallel group
Determine the appropriate Trulance (plecanatide) treatment dose for pediatric patients with chronic idiopathic constipation (CIC) who are 2 years to less than 6 years of age by assessing the safety and efficacy of once daily oral plecanatide in
Unless FDA agrees, based on your completion of Postmarketing Commitment Number 1 above, that it is not appropriate to conduct a pediatric study in pediatric patients age 12 to 17 years, conduct a pediatric study in pediatric patients age 12 to
A postmarketing prospective, long-term, observational study to assess the longterm safety of secukinumab compared to other therapies used in the treatment of adults with moderate to severe plaque psoriasis who are candidates for systemic therap
Complete the treatment and evaluation of subjects enrolled in the ongoing CAIN457A2302E1 trial for a duration of 4 years, unless a safety signal is identified that indicates the potential risks of such continued long-term treatment outweigh the
Complete the treatment and evaluation of subjects enrolled in the ongoing CAIN457A2304E1 trial for a duration of 4 years, unless a safety signal is identified that indicates the potential risks of such continued long-term treatment outweigh the
Conduct a clinical trial to assess whether secukinumab alters the metabolism or pharmacokinetics of CYP substrates in psoriasis patients treated with secukinumab.
The initiation of study CAIN457A2110 was delayed due to interactions between the Sponsor and the FDA in order to reach consensus on the study design. The revised schedule was acknowledged by FDA on September 29, 2015. The target enrollment is 2
Conduct a clinical trial to evaluate the treatment effect and safety profile of a higher exposure (e.g., 450 mg) of secukinumab in psoriasis subjects with higher body weight and to explore the option of exposure escalation (e.g., 450 mg) for th
The final protocol submission milestone date of 11/2015 was missed. Original protocol CAIN457A2324 was submitted 3/27/15 and discussions are currently ongoing. Initiation of CAIN457A2324 will begin upon FDA concurrence of the study design.
To develop and maintain a Pregnancy Surveillance Program that collects pregnancy and fetal outcomes of women exposed to romiplostim during pregnancy. Reports from the program will include an analysis of reports on major and minor congenital ano
A safety and pharmacokinetic trial as a sub-study of the trial described in PMR #5 above to evaluate trough concentrations of adalimumab and antibody levels (utilizing a validated anti-adalimumab antibody assay as described in PMR #3 above) at
Develop and validate anti-ecallantide and anti-P. pastoris-specific human IgE detection assays using a sensitive platform such as ECL.  Such assays should be free from interference by anti-ecallantide IgG antibodies.
Conduct a prospective, observational registry study of 100 rituximab-treated patients with Wegener¿s granulomatosis (WG) or microscopic polyangiitis (MPA) followed for 4 years to evaluate long term safety and retreatment with rituximab or othe
Conduct a trial to determine the efficacy and safety of the use of deferiprone to treat iron overload in patients with sickle cell disease and transfusional hemosiderosis who have not been adequately treated with available chelating agents. Sub
Establish a registry in order to perform an enhanced pharmacovigilance study of agranulocytosis. Procedures should include: Creation of marketing materials to inform and encourage clinicians to report agranulocytosis events to the sponsor; moni
Evaluate the long-term, prospective clinical outcomes of treatment with sebelipase alfa in adult and pediatric patients with LAL deficiency, including but not limited to progression of liver and cardiovascular diseases and changes in anthropome
Collect and analyze data related to the home administration of Synribo in order to assess the potential for the serious safety risks of incorrect dose administration and serious adverse events potentially arising from improper distribution, tra
Conduct an in vivo comet assay for [...]
Conduct a pre- and post-natal development toxicology study in the rat model to assess the potential impact of dibutyl sebacate on development.
Provide data on overall response rate with osimertinib from one or more “realworld” cohorts of a minimum of 100 patients who have been selected for treatment on the basis of an EGFR T790M mutation positive result from plasma (ctDNA) using t
Submit the final clinical report and datasets at the time of the final analysis for overall survival (OS) for Trial P006, entitled “A Multicenter, Randomized, Controlled, Three-Arm, Phase III Study to Evaluate the Safety and Efficacy of Two D
Develop and validate an appropriate analytical method for determining measurable amounts of the following 9 phytosterols in Intralipid 20% IV Fat Emulsion: sitosterol, desmosterol, brassicasterol, lanosterol, ergosterol, campesterol, stigmaster
Using the method developed under PMR 3109-1, test for the individual component phytosterol content in at least one batch of Intralipid 20% IV Fat Emulsion manufactured with every new lot of soybean oil over a three year period. Based on these t
Randomized controlled trial in pediatric patients, including neonates, comparing Intralipid 20% IV Fat Emulsion with a phytosterol-depleted formulation of Intralipid 20% IV Fat Emulsion to evaluate the incidence of liver injury, including eithe
A randomized, single-blind, multicenter dose-ranging study comparing the safety and efficacy of ColPrep Kit (sodium sulfate, potassium sulfate and magnesium sulfate) for oral solution to an active control or community standard of care in childr
ColPrep Kit® (Sodium Sulfate, Potassium Sulfate, and Magnesium Sulfate)
A randomized, single-blind, multicenter dose-ranging study comparing the safety and efficacy of ColPrep Kit (sodium sulfate, potassium sulfate and magnesium sulfate) for oral solution to an active control or community standard of care in childr
A two-year carcinogenicity study in one rodent species (CD-1 mice) with subcutaneous administration of nusinersen.
A pre-and postnatal development (including maternal function) study of nusinersen in rodent.
Conduct a clinical trial to test whether the addition of hormonal therapy increases the efficacy of pertuzumab-based therapy in the hormone receptor-positive, HER2-positive metastatic breast cancer population. Study MO27775 (PERTAIN) as designe
A medullary thyroid carcinoma case series registry of at least 15 years duration to systematically monitor the annual incidence of medullary thyroid carcinoma in the United States and to identify any increase related to the introduction of Tanz
Evaluate in a step-wise approach the disease-drug-drug interaction (Disease-DDI) potential for vedolizumab to indirectly affect the exposure of CYP substrate drugs by modulating pro-inflammatory cytokines in patients with ulcerative colitis and
To determine the potential for pharmacokinetic interactions between Erbitux (cetuximab) and cisplatin you will conduct the drug-drug interaction clinical trial, Study I4E-MC-JXBA.
Following the assessment of data from Trial CA184024, you will design and conduct a trial to compare the efficacy, with the primary endpoint of overall survival and the safety of ipilimumab at doses of 3mg/kg versus 10mg/kg given as monotherapy
Submit the final clinical report and datasets at the time of the final analysis for overall survival (OS) for Trial P002, entitled “Randomized, Phase 2 Study of MK-3475 versus Chemotherapy in Patients with Advanced Melanoma”, to revise the
A 54-week, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of JANUMET XR versus metformin extended-release in pediatric patients who are inadequately controlled on metformin immediate release.
Janumet® XR (Metformin Hydrochloride and Sitagliptin Phosphate)
A prospective, observational study designed to quantify the serious risks of misuse, abuse, and addiction associated with long-term use of opioid analgesics for management of chronic pain among patients prescribed ER/LA opioid analgesics. This
An observational study designed to measure the incidence and predictors of opioid overdose and death (OOD), as well as opioid abuse/addiction, using patient health records, insurance claims, and death records. This study must address at a minim
A prospective observational study designed to assess the content validity and patient interpretation of the Prescription Opioid Misuse and Abuse Questionnaire (POMAQ). Patient understanding of the concepts of misuse and abuse will also be obtai
Deferred 3-month cariprazine toxicity study in the juvenile rat starting at the appropriate age that corresponds to children age of 10 years. A dose range finding/toxicokinetic (TK) study should be conducted prior to a definitive toxicity/TK st
Deferred 6-month study in the juvenile dog starting at the appropriate age that corresponds to children age of 10 years. A dose range finding/TK study should be conducted prior to a definitive toxicity/TK study. TK assessment should include car
Deferred pediatric study under PREA (ages 10 to 17 years) with a diagnosis of schizophrenia or bipolar I disorder to obtain pharmacokinetic, safety, and tolerability data to inform the selection of doses in efficacy and safety studies in pediat
Deferred pediatric study under PREA for the treatment of schizophrenia in patients aged 13 to 17. A study of the efficacy and safety of cariprazine in the relevant pediatric population.
Deferred pediatric study under PREA for the treatment of bipolar I disorder, manic episodes in patients aged 10 to 17. A study of the efficacy and safety of cariprazine in the relevant population.
Deferred long-term, open-label safety study in pediatric patients with schizophrenia (ages 13 to 17) and bipolar I disorder, recent manic episodes (ages 10 to 17).
An in vivo drug-drug interaction study to assess cariprazine exposure when cariprazine is coadministered with a proton pump inhibitor.
In-vitro evaluation of: 1) inhibition potential of cariprazine, and the metabolites DCAR and DDCAR toward CYP2C8; 2) inhibition potential of DCAR and DDCAR toward CYP2B6 and CYP2C19; 3) induction potential of cariprazine , DCAR and DDCAR toward
Conduct a placebo-controlled, randomized withdrawal, dose-response trial in adults patients with schizophrenia to assess the long-term, dose-related serious adverse effects of cariprazine, including tardive dyskinesia, akathisia, adrenal dysfun
Conduct a placebo-controlled, randomized withdrawal, dose-response trial in adult patients with bipolar I disorder to assess the long-term, dose-related serious adverse effects of cariprazine, including tardive dyskinesia, akathisia, adrenal dy
A study to assess for the presence of antibodies that bind native double-stranded (ds) DNA among patients treated with Spinraza (nusinersen). The study may be conducted with plasma samples from patients treated with Spinraza (nusinersen) in the
Enhanced pharmacovigilance program for reports of malignancy in pediatric, adolescent, and young adult (<  30 years of age) patients treated with Simponi (golimumab), for a period of up to 10 years after this notification to collect data that w
Conduct a clinical trial to evaluate the impact of hepatic impairment on the pharmacokinetics of Kadcyla (ado-trastuzumab emtansine), total trastuzumab, and DM1-containing catabolites.   Based on the results of this trial, update the approved K
Deferred pediatric 12-month Open-Label Safety Study under PREA for prophylaxis of headaches in adolescents ages 12 to 17 with chronic migraine. The study must include at least 300 patients who received two BOTOX treatments at clinically relevan
Original Final Report Due Date: 09/30/2017; Deferral Extension granted per FDA letter dated 01/08/2015
Genzyme commits to performing additional analyses of the data obtained in the registry of patients with Fabry disease being treated with Agalsidase beta that was established to obtain long-term clinical status information.  Additional analyses
An observational study to develop and validate an algorithm using coded medical terminologies and other electronic healthcare data to identify opioid-related overdose and death.
An observational study to develop and validate an algorithm using coded medical terminologies to identify patients experiencing prescription opioid abuse or addiction, among patients receiving an ER/LA opioid analgesic.
An observational study using coded medical terminologies and other electronic healthcare data to define and validate doctor and/or pharmacy shopping outcomes by examining their association with abuse and/or addiction.
An observational study using a validated patient survey to evaluate the association between doctor/pharmacy shopping outcomes and self-reported misuse and abuse.
An observational study using medical record review to evaluate the association between doctor/pharmacy shopping outcomes and patient behaviors suggestive of misuse, abuse and/or addiction.
Conduct a clinical trial to estimate the serious risk for the development of hyperalgesia following the long-term use of high-dose ER/LA opioid analgesics for at least one year to treat chronic pain. Include an assessment of risk relative to ef
Conduct a study to evaluate safety and pharmacokinetics of Sernivo (betamethasone dipropionate) Spray, 0.05% versus Diprolene Lotion, 0.05% in pediatric subjects 12 years to 16 years 11 months of age with moderate to severe plaque psoriasis. As
Per FDA letter dated 10/06/2016, this PMR has been released.
Conduct a study to determine if Tysabri (natalizumab) increases the risk of major congenital malformations. In this study, the risk of fetal and infant outcomes (including malformations) among Tysabri-exposed women identified from the Tysabri P
To submit the clinical study report and datasets for the final analysis of overall survival (OS) for Trial CRAD001T2302, entitled “A randomized, double-blind, multicenter, phase III study of everolimus (RAD001) plus best supportive care versu
Submit the final study report for the stage 2 (GClb versus RClb) of the BO21004/CLL11 trial entitled, “An Open-label, Multi-center, Three Arm Randomized, Phase 3 Study to Compare the Efficacy and Safety of RO5072759 + Chlorambucil (GClb), Rit
Submit the final study report for the clinical trial GAO4753g (GADOLIN) entitled, “An open-label, multicenter, randomized, phase 3 study to investigate the efficacy and safety of bendamustine compared with bendamustine+RO5072759 (GA101) in pa
A randomized, double-blind, placebo-controlled clinical trial to evaluate the effect of testosterone replacement therapy on the incidence of major adverse cardiovascular events in men. We recommend that this trial also assess other important sa
A study in Fischer 344 rats to ascertain the effect of different Natpara (parathyroid hormone) dosing regimens on osteoblast proliferation, as an indicator of relative osteosarcoma risk.
An enhanced pharmacovigilance study of osteosarcoma in patients with hypoparathyroidism treated with Natpara (parathyroid hormone). The study will include reports of osteosarcoma for a period of 15 years from the date of approval, and will incl
A clinical pharmacology trial to assess the pharmacokinetics (PK) and pharmacodynamic effects (PD) of Natpara (parathyroid hormone) dose and dosing regimen on the control of serum calcium and normalization of calcium excretion in urine. Modelin
The Final Protocol Submission milestone was missed because of ongoing discussion with FDA regarding study design. FDA determined that the applicant demonstrated good cause for the delay.
A 26-week randomized, controlled clinical trial to evaluate the longer term safety and effect of an alternative dose(s) and/or dosing regimen(s) of Natpara (parathyroid hormone), including longer term safety with respect to hypercalciuria. This
Develop and test the stability of a lower (35 or 70 mg) strength ibrutinib capsule in order to allow dose reductions for patients with moderate hepatic impairment for whom ibrutinib treatment is currently not recommended. The lower strength cap
Protocol was due 9/2015. Sponsor did not submit protocol to the IND.
Evaluate the pharmacokinetics, safety and treatment response (using sustained virologic response as the primary endpoint) of ombitasvir, paritaprevir, ritonavir,dasabuvir (Viekira Pak™) in pediatric subjects 3 to less than 18 years of age wit
Original Final Report Due Date: 08/31/2019; Deferral Extension granted per FDA letter dated 07/08/2016.
Viekira Pak® (Ombitasvir, Paritaprevir, and Ritonavir; Dasabuvir)
Collect and analyze long-term safety data for subjects enrolled in the pediatric ombitasvir, paritaprevir, ritonavir, dasabuvir (Viekira Pak™) pharmacokinetic, safety, and antiviral efficacy study(ies). Data collected should include at least
Original Final Report Due Date: 08/31/2022; Deferral Extension granted per FDA letter dated 07/08/2016.
Submit a final report for ongoing observational study M13-102, "A Follow-up Study to Assess Resistance and Durability of Response to AbbVie Direct-Acting Antiviral Agent (DAA) Therapy in Subjects Who Participated in Phase 2 or 3 Clinical Studie
Conduct an observational study to investigate the safety and efficacy of ombitasvir, paritaprevir, ritonavir, dasabuvir (Viekira Pak™) in a sufficient number of Blacks/African Americans with and without cirrhosis compared to whites/Caucasians
Submit the final report and datasets for the ongoing clinical trial M14-227 entitled "An Open-Label Study to Evaluate the Safety and Efficacy of ABT- 450/Ritonavir/ABT-267 and ABT-333 with Ribavirin in Adults with Genotype 1 Chronic Hepatitis C
To test all banked clinical samples from pivotal clinical trials NIH 991265/20010769 and trial FHA101 for the presence of neutralizing antibodies against leptin using the ligand binding assay developed and validated under PMR#2, and to correlat
The final report milestone was missed, because sample testing was delayed due to assay development and time required to reconsent patients
To provide an update of efficacy from all 3 treatment arms in Study BCIRG006 at the time when the last patient enrolled reaches 10 years of follow-up, with an interim update of efficacy at 5-years of follow-up.
Conduct a field study to evaluate the efficacy, pharmacokinetics, and safety of raxibacumab use for Bacillus anthracis in the United States.
Conduct a PK/safety study of SC abatacept in polyaricular JIA patients ages 6 to 17 years of age.
Enrollment for 6-17 year olds completed as of 12/22/2015. Enrollment completion for 2-5 year olds targeted for 2Q2016.
Conduct a prospective, observational study utilizing data from the United Network for Organ Sharing (UNOS) on the pattern of belatacept use in US adult kidney-only transplant recipients at transplant and one year post-transplant.  Specifically,
A single-center randomized, placebo-controlled and active-controlled thorough QT (TQT) trial in normal (or healthy) subjects. Please refer to ICH E14 guidance to design the trial and submit the protocol to the agency for comments. The doses stu
The sponsor submitted on 11/23/15 proposed revised milestones dates due to their protocol study design negotiations with the agency.
Conduct a dose-ranging Pharmacokinetics (PK), Safety and Efficacy Study in pediatric subjects 6 to <18 years of age with moderate to severe psoriasis (with a duration of exposure to ixekizumab of at least one year).
Conduct a retrospective cohort study using administrative databases to identify pregnancy outcomes in a cohort of women exposed to ixekizumab and a nonixekizumab systemic medication exposure cohort. The outcomes will include major congenital ma
Conduct a prospective registry-based observational exposure cohort study that compares the maternal, fetal, and infant outcomes of women exposed to ixekizumab during pregnancy to an unexposed control population. The registry will detect and rec
Conduct a prospective, observational study to assess the long-term safety of ixekizumab compared to other therapies used in the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy
Conduct a clinical study to assess whether ixekizumab alters the metabolism or pharmacokinetics of CYP substrates in psoriasis patients treated with ixekizumab.
Conduct a Field Study to evaluate the clinical response, pharmacokinetics, and safety profile of Anthim (obiltoxaximab) when used in the treatment of suspected or confirmed cases of inhalational anthrax due to B. anthracis in the United States.
In patients with CKD who are not on dialysis (NOD), conduct one or more trials to determine whether a dosing strategy (e.g. fixed dose strategy) different from that in the approved labeling can further reduce exposure to ESA while preserving th
Submit the final report and datasets for the ongoing clinical trial M14-226 entitled "An Open-Label Study to Evaluate the Safety and Efficacy of Ombitasvir/ABT- 450/Ritonavir and Dasabuvir with or without Ribavirin (RBV) in Treatment- Naïve Ad
Submit the final report and datasets for the ongoing Phase 3 clinical trial M11-646 entitled "A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of ABT-450/Ritonavir/ABT-267 (ABT- 450/r/ABT-267) and ABT-333
Submit the final report and datasets for the Phase 3 clinical trial M13-098 entitled "A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of ABT-450/Ritonavir/ABT-267 (ABT-450/r/ABT-267) and ABT-333 Co-admin
Submit the final report and datasets for the Phase 3 clinical trial M13-099 entitled "A Randomized, Open-Label Study to Evaluate the Safety and Efficacy of ABT- 450/Ritonavir/ABT-267 (ABT-450/r/ABT-267) and ABT- 333 Coadministered with Ribaviri
Submit the final report and datasets for the Phase 3 clinical trial M13-961 entitled "A Randomized, Double-Blind, Controlled Study to Evaluate the Efficacy and Safety of the Combination of ABT-450/Ritonavir/ABT-267 (ABT-450/r/ABT- 267) and ABT-
Submit the final report and datasets for the Phase 3 clinical trial M13-389 entitled "A Randomized, Open-Label, Multicenter Study to Evaluate the Safety and Antiviral Activity of the Combination of ABT-450/Ritonavir/ABT-267 (ABT- 450/r/ABT-267)
Submit the final report and datasets for the Phase 3 clinical trial M14-002 entitled "A Randomized, Double-Blind, Controlled Study to Evaluate the Efficacy and Safety of the Combination of ABT-450/Ritonavir/ABT-267 (ABT-450/r/ABT- 267) and ABT-
Submit the final report and datasets for the ongoing clinical trial M12-999 entitled "Open-label, Phase 2 Study to Evaluate the Safety and Efficacy of the Combination of ABT-450/ritonavir/ABT-267 (ABT-450/r/ABT-267) and ABT- 333 With or Without
Submit the final report and datasets for the ongoing clinical trial M14-004 entitled "A Randomized, Open-label Study to Evaluate the Safety and Efficacy of ABT- 450/Ritonavir/ABT-267 (ABT-450/r/ABT-267) and ABT-333 Coadministered with Ribavirin
Submit the final report and datasets for the ongoing clinical trial M14-490 entitled "An Open-Label, Single-Arm Study to Evaluate the Safety and Efficacy of Ombitasvir/ABT-450/Ritonavir and Dasabuvir in Adults with Genotype 1b Chronic Hepatitis
A 6-month carcinogenicity study in the transgenic mouse. Submit the carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study.
A long-term rodent carcinogenicity study in the rat. Submit the carcinogenicity protocol for a Special Protocol Assessment (SPA) prior to initiating the study.
Provide quarterly reports on the status of any [ ... ]. These reports should include, at a minimum, a summary of the root cause analyses, associated corrective actions, and disposition of all affected DM1 batches.  Also, provide the disposition
A single-center randomized, placebo-controlled and active-controlled thorough QT (TQT) trial of Remeron (mirtazapine) in normal (or healthy) subjects. Please refer to ICH E14 guidance to design the trial and submit the protocol to the agency fo
MERCK SHARP AND DOHME CORP A SUBSIDIARY OF MERCK AND CO INC
A single-center, randomized, placebo-controlled and active-controlled thorough QT (TQT) trial of Effexor Extended-Release (venlafaxine hydrochloride) in normal (or healthy) subjects. Please refer to ICH E14 guidance to design the trial and subm
The FDA is currently reviewing the protocol for Study B2411360 submitted on 19 November 2015. The projected first subject dose is for 6 January 2016. The timeline for this PMR is currently undergoing renegotiation with the FDA. Pfizer has propo
WYETH PHARMACEUTICALS INC A SUBSIDIARY OF PFIZER INC
An open label pharmacokinetic and safety study of SPRIX in pediatric patients ages 12 to less than 17 years of age
S-010 submitted on June 18, 2015, but UF letter issued due to unpaid user fee
An open label pharmacokinetic and safety study of SPRIX in pediatric patients ages 2 to less than 12 years of age
Supplement submitted, but FDA did not file.
A pharmacokinetic, safety, and efficacy study of SPRIX in pediatric patients aged 1 month to less than 2 years of age.
Complete Clinical Trial C13008, an open-label trial to determine the long-term safety of Entyvio (vedolizumab) in patients with ulcerative colitis and Crohn’s disease.  Safety evaluations include but are not limited to the occurrence of serio
Conduct a prospective, observational pregnancy exposure registry study in the United States that compares the pregnancy and fetal outcomes of women exposed to Entyvio (vedolizumab) during pregnancy to an unexposed control population or collect
Conduct an open-label trial to assess the clinical significance of the drug-drug interaction between a single 1200 mg IV dose of Orbactiv (oritavancin diphosphate) and warfarin in healthy volunteers.
Final study report was due in June, 2015, revised to November, 2015
Conduct an open label trial evaluating the safety of a single 1200 mg IV dose of Orbactiv (oritavancin diphosphate) in patients on concomitant chronic warfarin therapy who are being treated for ABSSSI.
Conduct a single-center, open-label trial to evaluate the effects of a single 1200 mg IV dose of Orbactiv (oritavancin diphosphate) on the results of multiple coagulation tests in healthy volunteers.
Conduct a study to evaluate the effects of oritavancin on phospholipid and nonphospholipid based coagulation tests in vitro.
Conduct a clinical trial to evaluate the effect of renal impairment on eliglustat pharmacokinetics. A reduced design may be used.
Conduct a clinical trial to evaluate the effects of hepatic impairment on eliglustat pharmacokinetics.
Continue to follow patients en rolled in trial CC-5013-MCL-001 for at least 4 years from the date that the last patient enroll ed. Submit a report to the Agency describing the cumulative efficacy and safety data up to this 4 year time point.
Analyze the polyethylene oxide (PEO) product employed in Zohydro ER (hydrocodone bitartrate) for low molecular weight impurities. Identify and quantitate the impurities. Submit a toxicological risk assessment for the exposure to the impurities
Conduct an embryo-fetal development study in the rat model to assess the potential impact of polyethylene oxide (PEO) on development. The study must be designed to adequately qualify the safety of the low molecular weight PEO components (impuri
Conduct an embryo-fetal development study in the rabbit model to assess the potential impact of polyethylene oxide (PEO) on development. The study must be designed to adequately qualify the safety of the low molecular weight PEO components (imp
Conduct a pre- and post-natal development study in the rat model to assess the potential impact of polyethylene oxide (PEO) on development. The study must be designed to adequately qualify the safety of the low molecular weight PEO components (
Conduct a pharmacokinetic, pharmacodynamics, tolerability and dose ranging study in subjects 6 years through 17 years, to assess safety and to determine the doses to be used in the efficacy study. Age-specific endpoints, i.e. a clinical outcome
Conduct a randomized, controlled, double-blind study to determine the safety and efficacy of rifaximin in pediatric patients 6 years through 17 years with diarrhea-predominant irritable bowel syndrome (IBS-D).
Conduct in vitro studies to properly determine the IC50 values for the inhibition of OATP1B1, OATP1B3, and OATP1A2 by rifaximin. The in vitro study results will determine the need for subsequent clinical assessments of drug interactions by rifa
Conduct a dose ranging study to determine the safety and effectiveness of eluxadoline in pediatric patients 6 through 17 years with diarrhea-predominant irritable bowel syndrome (IBS-D). The pharmacokinetics of eluxadoline in these pediatric pa
The final protocol submission milestone was missed.
Conduct a randomized, double-blind study to determine the safety and effectiveness of eluxadoline in pediatric patients 6 through 17 years with diarrheapredominant irritable bowel syndrome (IBS-D).
Conduct an open-label extension safety study of eluxadoline in pediatric patients 6 through 17 years with diarrhea-predominant irritable bowel syndrome (IBS-D) who participated in the dose ranging (# 2901-1) or efficacy (# 2901-2) studies.
A dedicated clinical pharmacology trial to evaluate the impact of renal impairment on eluxadoline pharmacokinetics and the risk for euphoria and other central nervous system (CNS) adverse effects.
A descriptive observational cohort study to evaluate performance of the THI-002 inserter in women receiving Liletta. The study cohort will include a minimum of 1,000 women who receive Liletta using the THI-002 inserter in a variety of clinical
A Pregnancy Pharmacovigilance Study to evaluate pregnancy outcomes and infant outcomes following exposure to Odomzo (sonidegib). This study will include a mechanism to collect, classify, and analyze data on direct exposures (women exposed to Od
Complete the ongoing pharmacokinetic trial to determine an appropriate dose of Odomzo (sonidegib) in patients with moderate to severe hepatic impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics in Patients w
Submit the Final Report for the clinical pharmacokinetic (drug interaction) trial to determine how to dose a gastric acid-reducing agent with Odomzo (sonidegib).
Conduct an in vitro study evaluating interactions between dalbavancin hydrochloride and coagulation tests.
Conduct a pharmacoepidemiology study (Protocol IM101045A) to assess the short term (2 years) and potential long-term (4 years) risk of hospitalization due to infection (all hospitalized infections, hospitalized pneumonia, and all opportunistic
To submit an efficacy supplement containing the final study report, including summary analyses and datasets and revised labeling based on the results of study AVF4396g/BO20990 entitled "A Randomized, Double Blind, Placebo Controlled, Multicente
Conduct a 12-week, randomized, double-blind, double-dummy, parallel group, placebo-controlled, dose-ranging, efficacy, and safety study in children 5-11 years of age with asthma. The final study report will be submitted as a supplement with the
421/596 subjects completed the study. Study is completed, pending final study report.
GLAXOSMITHKLINE INTELLECTUAL PROPERTY DEVELOPMENT LTD ENGLAND
Conduct a 2-week randomized, double-blind, placebo-controlled, 2-way crossover, knemometry growth rate study in children 5-11 years of age with asthma.
58/60 subjects completed the study. Study is completed, pending final study report.
Conduct a 52-week, randomized, double-blind, parallel group, active controlled, growth study in females 5-<8 years of age and males 5-<9 years of age with asthma.
Conduct a 6-week, randomized, double-blind, parallel group, placebo-controlled, HPA-axis study in children 5-11 years of age with asthma.
111 subjects enrolled. Study is completed, pending final study report.
A PK-PD bridging simulation study must be conducted to determine the appropriate lacosamide monotherapy regimen for the treatment of partial-onset seizures in the pediatric population > or equal to 1 month to < 17 years of age, and to support e
An open-label safety and tolerability study using lacosamide as monotherapy in pediatric patients > or equal to 1 month to < 17 years of age.
A safety study of replacement of oral dosing with intravenous dosing administered over 30 to 60 minutes in pediatric patients > or equal to 1 month to < 17 years of age with partial-onset seizures. If safety is acceptable, a replacement study a
A study that will examine safety and tolerability of an oral loading dose that will allow a more rapid achievement of the final recommended therapeutic dose in pediatric patients ¡Ý 1 month to < 17 years of age.
A study that will examine safety and tolerability of an intravenous loading dose that will allow a more rapid achievement of steady-state exposures of the final recommended therapeutic dose in pediatric patients ¡Ý 1 month to < 17 years of ag
Prospective, randomized (1:1), open-label, parallel group, active controlled, multicenter study to assess safe and effective doses of Kabiven (amino acids, electrolytes, dextrose and lipid injectable emulsion) in pediatric patients aged 2 to 16
Deferral Extension Requested 11/10/2016.  Denied per FDA letter dated 12/20/2016.
To conduct an animal study that will measure the effect of PD-1 inhibition on the magnitude of the primary (first vaccination) and recall (second vaccination) antibody responses to antigen challenge (e.g., tetanus toxoid or KLH). This study wil
A safety assessment of deoxycholic acid treatment in subjects aged 65 years and older. This assessment is to be performed in the ongoing ATX-101-13-28 trial population of subjects aged 65 to 75 years. To the extent possible, all subjects should
Conduct a randomized trial that will characterize the incidence, severity and response to treatment of nivolumab induced immune-mediated adverse reactions to include immune-mediated pneumonitis.
Submit the final report and efficacy datasets for the open-label randomized trial of nivolumab versus docetaxel in patients with previously treated advanced squamous non-small cell lung cancer.
Deferred pediatric assessment under PREA for Truvada (emtricitabine/tenofovir disoproxil fumarate) for the treatment of HIV-1 infection in pediatric subjects from ages 6 years to less than 12 years, weighing at least 17 kg.
Per FDA letter dated 03/10/2016, this PMR has been fulfilled.
A drug interaction trial to determine the effects of sorbitol-containing solutions on lamivudine exposure following administration of lamivudine oral solution. Evaluate bioavailability differences between lamivudine solution administered alone
A multi-center clinical trial to evaluate the dosing and safety of PA-21 for the treatment of hyperphosphatemia in children ages 1 month to 17 years with chronic kidney disease (CKD) stages 4-5 (defined by a glomerular filtration rate <30 mL/mi
Protocol was submitted 31-Oct-2014.Protocol amendment submitted 02-Apr-2015
VIFOR FRESENIUS MEDICAL CARE RENAL PHARMA FRANCE
To commit to testing samples from patients treated with Oncaspar for the presence of binding and neutralizing antibodies to Oncaspar using the validated assays discussed in items 1 and 2 in the following study, entitled, "A Multi-Center, Open L
Study terminated because the initial dose level tested exceeded the MTD. Report submitted September 15, 2009.
Complete the trial and submit the final report and data to verify and describe the clinical benefit of blinatumomab, including efficacy and safety from Protocol 00103311, a Phase 3 randomized, open-label, active-controlled trial comparing blina
A randomized, controlled, parallel group superiority trial in pediatric patients ages 10 through 17 years to evaluate the safety and efficacy of Plegridy (peginterferon beta-1a) compared to an appropriate control for the treatment of relapsing
A prospective, observational, exposure cohort study conducted in the United States that compares the maternal, fetal, and infant outcomes of women with multiple sclerosis exposed to Plegridy (peginterferon beta-1a) during pregnancy to unexposed
The final protocol milestone was missed, because the final protocol is still under negotiation.
Conduct a trial to evaluate pediatric pharmacokinetics (PK), safety and antiviral activity of once daily atazanavir and cobicistat (ATV/COBI) combined with a background regimen in HIV-1 infected pediatric subjects. Subjects receiving ATV/COBI s
A clinical trial in healthy subjects evaluating the effect of cobicistat coadministered with atazanavir at steady state on the pharmacokinetics of atorvastatin.
A clinical trial in healthy subjects evaluating the effect of cobicistat coadministered with atazanavir at steady state on the pharmacokinetics of rosuvastatin.
A clinical trial in healthy subjects evaluating the effect of cobicistat coadministered with atazanavir at steady state on the pharmacokinetics of the estrogen and progestin components of a combined oral contraceptive.
In patients with CKD on dialysis, conduct one or more trials to identify an optimal strategy of ESA dose and schedule. These trials should identify the optimal dosing strategy which will demonstrate the superiority of the ESA dosing strategy to
A juvenile animal study with the combination of bupropion and naltrexone to assess post-natal growth and development with additional assessment of behavior, learning and memory.
Conduct a clinical trial designed to determine whether a higher dose of ramucirumab, in combination with paclitaxel, provides superior progression-free survival as compared to the recommended ramucirumab dose in combination with paclitaxel, in
Conduct the analysis and submit the complete final report and data showing clinical efficacy and safety from Trial MMY3003, a “Phase 3, 2- arm, Randomized, Parallel-group Trial of Lenalidomide and Dexamethasone with or without Daratumumab in
Conduct the analysis and submit the complete final report and data showing clinical efficacy and safety from Trial MMY3004, a “Phase 3, 2- arm, Randomized, Parallel-group Trial of Bortezomib and Dexamethasone with or without Daratumumab in Pa
Submit the final report of a study conducted to assess the anti-drug antibody (ADA) response to daratumumab with the validated assay developed under PMR 3000-4
Conduct a study to validate an assay for binding antibodies to daratumumab to assess the product’s potential for immunogenic reactions in treated patients. Submit a validation report for the validated, sensitive, and accurate assay for the de
Conduct a study to validate an assay for neutralizing antibodies to daratumumab to assess the potential for increased adverse outcome from loss of product effect in treated patients. Submit a validation report for the validated, sensitive, and
The data included in the final report submission does not adequately fulfill the postmarketing requirement.The Original Final Report due date was not met.
Collect, analyze, and submit additional safety data from ongoing clinical trials to characterize the safety of daratumumab in patients with baseline hepatic impairment.
Establish the optimal and safe dose of pralatrexate in combination with the cyclophosphamide/vincristine/doxorubicin/prednisone (CHOP) regimen. Perform a Phase 1 dose-finding trial of pralatrexate plus CHOP for the treatment of patients with pe
The final report has not been submitted and the clinical trial completion date milestone has been missed.
Characterize the comparative efficacy and safety of pralatrexate when used in combination with a treatment regimen such as CHOP versus the combination of Beleodaq plus CHOP, versus CHOP alone for the initial therapy of patients with PTCL. Perfo
Conduct an open-label, single-arm trial to evaluate the long-term safety of Kalydeco (ivacaftor) in pediatric patients with Cystic Fibrosis (CF) and R117H mutation in the CFTR. The trial will enroll pediatric patients < 18 years of age and who
An open-label pharmacokinetic (PK), and multiple-dose safety and tolerability dose-titration trial of Afrezza in pediatric patients ages 4 to 17 years (inclusive) with type 1 diabetes (Part 1), followed by a prospective, multicenter, open-label
Conduct a dose-ranging PK-PD euglycemic glucose-clamp trial to characterize the dose-response of Afrezza relative to subcutaneous insulin in patients with type 1 diabetes. Select at least three to four doses for each route of insulin administra
A PK-PD eugylcemic glucose-clamp trial to characterize within-subject variability for Afrezza pharmacokinetic (PK) and pharmacodynamic (PD) parameters. These data may impact labeling recommendations for glucose monitoring and thereby mitigate t
Conduct a 5-year, randomized, controlled trial in 8,000-10,000 patients with type 2 diabetes to assess the serious potential risk of pulmonary malignancy with Afrezza use. The primary objective of the trial should be to compare the incidence of
The Final Protocol Submission milestone was missed, because of ongoing discussion with FDA regarding the study design and FDA determined that the applicant demonstrated “good cause” for the delay.
Conduct a prospective study over a five-year period after the introduction of ZERBAXA (ceftolozane/tazobactam) to the market to determine if decreased susceptibility to ZERBAXA (ceftolozane/tazobactam) is occurring in the target population of b
Submit the progression-free survival (PFS) and overall survival (OS) analyses with datasets from clinical trial D0818C00002, SOLO-2, the ongoing randomized doubleblind, placebo-controlled, multi-center trial to assess the efficacy of olaparib m
Submit the progression-free survival (PFS) and overall survival (OS) analyses with datasets from clinical trial D0816C00010, a randomized trial establishing the superiority of olaparib over physician’s choice single-agent chemotherapy in the
Randomized,-double-blind, adequately controlled, multiple fixed doses, parallel group clinical trials of Myobloc (botulinum toxin Type B) in the following populations: botulinum toxin-naive children age 2-17 years with lower extremity spasticit
Identify the in vitro metabolic pathways of dichlorphenamide and characterize the potential for P450-mediated drug interactions due to inhibition or induction of these pathways in vitro. Refer to the draft Guidance for Industry: Drug Interactio
Conduct a trial to characterize the pharmacokinetics of dichlorphenamide in healthy volunteers, to include a single ascending dose assessment and a multiple ascending dose assessment, using a dose range of 25 to 200 mg. Pharmacokinetic paramete
Characterize safety of long-term use in patients treated with Kyprolis (carfilzomib) 20/56 mg/m2 plus dexamethasone. Submit a final report and datasets with safety and efficacy outcomes of current clinical trial 2011-003 (ENDEAVOR) with at leas
Characterize the comparative safety and efficacy of the 20/27 mg/m2 and the 20/56 mg/m2 regimens of carfilzomib. Submit a study report with safety and efficacy outcomes of SWOG Protocol S1304 and your analysis of what clinical parameters might
Post-marketing observational study to evaluate the risk for venous thromboembolic events (VTEs) with Lybrel (ethinyl estradiol 20 mcg/levonorgestrel 90 mcg). This study shall evaluate the risk for VTE with Lybrel use compared to an appropriate
Conduct a study to evaluate the safety of SITAVIG in pediatric patients greater than or equal to 10 years of age to less than or equal to 17 years of age with recurrent herpes labialis and to assess duration of HSV episode in the treated popula
Submit safety data assessing distant spread of toxin effects after multiple administrations of Botox (onabotulinum toxin A), during a minimum period of 12 months, collected in at least 100 pediatric patients (ages 2-17 years) and 100 adult pati
The final report milestone was missed, because the FDA was engaged in discussion with the applicant regarding the study design at the time the final report submission milestone was due and FDA determined that the applicant  demonstrated good ca
A juvenile rat toxicology study under PREA to identify the unexpected serious risk of adverse effects of Botox on postnatal growth and development. The study should utilize animals of an age range and stage(s) of development that are comparable
Original Final Report Due Date: 12/31/2011; Deferral Extension granted per FDA letter dated 08/25/2015.
Collect and analyze all cases of acute myelogenous leukemia/ myelodysplastic syndrome identified in patients treated with Lynparza (olaparib) on an annual basis. These interim reports should summarize all cases identified up until that reportin
Submit the final report for trial D0816C00006 entitled, “An Open-label, Non-randomized, Multicenter, Comparative, and Phase 1 Study of the Pharmacokinetics, Safety and Tolerability of Olaparib Following a Single Oral 300 mg Dose to Patients w
Submit the final report for trial D0816C00005 entitled, “An Open-label, Non-randomized, Multicenter, Comparative, Phase 1 Study to Determine the Pharmacokinetics, Safety and Tolerability of Olaparib Following a Single Oral 300 mg Dose to Pati
The interim report milestone was missed because of difficulties in accruing patients and FDA determined that the applicant demonstrated “good cause” for the delay.
Evaluate the pharmacokinetics, safety, and antiviral activity (efficacy) of atazanavir and cobicistat fixed-dose combination (FDC) age-appropriate formulation in HIV-infected pediatric subjects 3 months to less than 3 years of age. The safety a
Conduct a prospective study over a five-year period to determine if decreased susceptibility to Cresemba (isavuconazonium sulfate) is occurring in the target population of organisms that are in the approved Cresemba (isavuconazonium sulfate) la
Conduct a two-year mouse carcinogenicity study.
Conduct a two-year rat carcinogenicity study
Establish a registry to collect and analyze clinical efficacy-related outcome data on patients treated with isavuconazonium sulfate who have invasive mucormycosis or infection with non-fumigatus aspergillus species.
Conduct a clinical trial to evaluate the incidence of serious and severe (i.e. ¡Ý Grade 3) adverse reactions of an oral starting dose of 20 mg or of 14 mg daily compared to the 24 mg starting dose, with a comparable objective response rate. S
Conduct a study to compare exposure and safety data from approximately 220 patients who complete treatment with dinutuximab, pooling across dinutuximab lots and by individual lot, with the historical experience observed in approximately 1100 pa
Conduct a single-dose pharmacokinetic study in which the primary objective is to identify the dose(s) of codeine phosphate and chlorpheniramine maleate ER tablet that results in exposures of codeine and chlorpheniramine in children and adolesce
Tuxarin™ ER (Codeine Phosphate and Chlorpheniramine Maleate)
Conduct an open-label, multi-dose safety and tolerability study in children and adolescents 12 to 17 years of age. The population eligible for the study would be children and adolescents with cough/cold symptoms for whom a combination product t
A prospective, randomized, controlled, double-blinded, efficacy and safety study of brivaracetam for the adjunctive treatment of partial onset seizures in children from 1 month to less than 4 years of age. The primary efficacy endpoint during t
Long-term safety study of brivaracetam in the adjunctive treatment of partial onset seizures in children from 1 month to less than 16 years of age. Routine safety measures should be monitored. Behavioral and cognitive endpoints should be includ
Submit the final overall survival analysis with datasets from Trial A5481023, PALOMA-3 “A double-blind, phase III trial of fulvestrant with or without palbociclib in pre- and post-menopausal women with hormone receptor-positive, HER2-negative
Determine the change in overall past 30-day abuse and abuse via specific routes of administration (ROA), including swallowing intact, chewing and swallowing, dissolving and swallowing, snorting, smoking, and injecting, among individuals being a
Determine the change in the rate of poison center exposure calls, using the Researched Abuse Diversion and Addiction-Related Surveillance (RADARS®) System Poison Center Program. Exposure calls should be grouped as follows: (1) Intentional abus
Conduct a comparative single-dose pharmacokinetic and safety trial of azilsartan between infants, children, and adolescents with hypertension and healthy adults. Using data from the PK and safety trial, conduct an efficacy and safety, dose-find
Conduct an in vivo comet assay in liver to evaluate the potential genetic toxicology of hydrocodone.
Conduct a 2-year bioassay in the rat model to evaluate the carcinogenic potential of hydrocodone.
Conduct a 2-year bioassay in the mouse model to evaluate the carcinogenic potential of hydrocodone.
Deferred study under PREA to evaluate the pharmacokinetics of dexlansoprazole, maintenance of healing, and symptoms of endoscopy-proven erosive esophagitis (EE) in patients 1 year to 11 years of age.
Deferred study under PREA to evaluate the pharmacokinetics of dexlansoprazole, maintenance of healing, and symptoms of endoscopy-proven erosive esophagitis (EE) in patients 12 years to 17 years of age.
Deferred pediatric study under PREA for treating heartburn associated with nonerosive gastroesophageal reflux disease (GERD) in pediatric patients aged 1 year to 11 years.
Deferred pediatric study under PREA for treating heartburn associated with nonerosive gastroesophageal reflux disease (GERD) in pediatric patients aged 12 year to 17 years.
Deferred study under PREA to evaluate the long-term safety of dexlansoprazole for the maintenance of healing of erosive esophagitis (EE) in pediatric patients 1 month through 11 months of age, who require chronic treatment with dexlansoprazole
Deferred study under PREA to evaluate the long-term safety of dexlansoprazole for the maintenance of healing of erosive esophagitis (EE) in pediatric patients 1 year through 17 years of age, who require chronic treatment with dexlansoprazole du
Conduct a trial in moderately to severely active ulcerative colitis patients to evaluate the safety of induction regimens of adalimumab at doses higher than 160/80 mg. In this trial, the efficacy of Humira (adalimumab) should also be assessed,
Deferred pediatric study under PREA: Conduct a pharmacokinetic and safety study of an age-appropriate formulation of Hysingla ER in patients from ages 12 to less than 17 years with pain severe enough to require daily, around-theclock, long-term
The applicant has not reported the status.
Conduct an adequate extractable/leachable safety assessment for the [...] gray [...] rubber stopper used in your container closure system. This assessment must include controlled extraction studies to qualitatively and quantitatively determine
Conduct a field study to evaluate the efficacy and safety of moxifloxacin hydrochloride in the event of an attack with the intentional release of Y. pestis in the United States.
Submit data to assess the pharmacokinetics and safety of Avelox (moxifloxacin hydrochloride) Tablets and IV in pediatric patients 3 months to less than 18 years of age.
Per FDA letter dated 08/15/2016, this PMR has been fulfilled.
Determine the change in the prevalence of self-reported past 30-day abuse of OxyContin and agreed-upon comparators using the The Researched Abuse Diversion and Addiction-Related Surveillance (RADARS®) System Treatment Center Program (Opioid Tr
Determine the change in the incidence of non-fatal and fatal overdose associated with OxyContin exposure relative to the change associated with exposure to appropriate comparators using electronic healthcare data with linkage to an appropriate
Conduct an analysis of safety in a randomized, open-label multi-center clinical trial comparing defibrotide versus best supportive care in the prevention of hepatic veno-occlusive disease in adult and pediatric patients, including all adverse e
Design and conduct a study and provide the full final report and data sets to evaluate the safety and efficacy of ProvayBlue (methylene blue) for the treatment of methemoglobinemia. The minimal efficacy endpoints should include achieving a 50%
Conduct a clinical trial (or trials) to determine the extent of in vivo drug-drug interaction of ProvayBlue (methylene blue) as a modulator of CYP450 enzyme activity using CYP450 enzyme substrates.
Conduct a clinical trial to determine the effect of hepatic impairment on the pharmacokinetics and safety of ProvayBlue (methylene blue).
Conduct a clinical trial to evaluate the effect of varying degrees of renal impairment on the pharmacokinetics and safety of ProvayBlue (methylene blue) in subjects with renal impairment.
Combine all available pharmacokinetics (PK) data from different patient populations and healthy subjects in an integrated population PK model to evaluate the potential impact of tumor types on the PK of cabozantinib.
Complete the trial and submit the final report for the pediatric pharmacokinetic trial entitled “Pharmacokinetics of SFP iron delivered via dialysate in pediatric patients with chronic kidney disease on hemodialysis.”
Conduct a randomized withdrawal trial comparing pimavanserin 34 mg/day to placebo.
Conduct a randomized placebo-controlled trial or trials with predominantly frail and elderly subjects that would involve exposure of at least 500 subjects to pimavanserin 34 mg daily for a minimum of 8 weeks.
A study in the greater than or equal to 12- to less than 17-year old age group to evaluate the pharmacokinetics, efficacy, and safety of different doses of phenylephrine hydrochloride injection in patients undergoing general anesthesia and/or n
Conduct a fertility and early embryonic development toxicology study in the rat model for phenylephrine hydrochloride.
Conduct an embryo-fetal developmental toxicology study using the rat model for phenylephrine hydrochloride.
Conduct an embryo-fetal developmental toxicology study using the rabbit model for phenylephrine hydrochloride.
Conduct a peri- and post-natal developmental toxicology study in the rat model for phenylephrine hydrochloride.
Submit the progression free survival (PFS) and overall survival (OS) data and results from the ongoing Trial A5481008, PALOMA-2, “A Randomized, Multicenter, Double-blind Phase 3 Study of PD-0332991 (Oral CDK 4/6 Inhibitor) Plus Letrozole Vers
Submit the final report for your clinical trial A5481013 entitled, “A phase 1, open-label, single dose, parallel-group study to evaluate the pharmacokinetics of palbociclib (PD-0332991) in subjects with impaired hepatic function,” to assess
Submit the final report for your ongoing drug interaction trial (A5481039) entitled, “A phase 1, open-label, fixed-sequence, 2-cohort, 2-period study to investigate the effect of modafinil and pioglitazone given as multiple doses on single do
Conduct analysis from the ongoing Trial A5481008, PALOMA-2, “A Randomized, Multicenter, Double-blind Phase 3 Study of PD-0332991 (Oral CDK 4/6 Inhibitor) Plus Letrozole Versus Placebo Plus Letrozole for the Treatment of Postmenopausal Women w
Conduct a randomized Phase 2 clinical trial of panobinostat in combination with subcutaneous bortezomib and dexamethasone to characterize the safety and efficacy of at least two different doses of panobinostat. Eligible patients will include pa
Conduct a multicenter, randomized, placebo-controlled Phase 3 trial comparing panobinostat in combination with subcutaneous bortezomib and dexamethasone with subcutaneous bortezomib and dexamethasonein patients with relapsed multiple myeloma wh
Conduct a single-dose pharmacokinetic study whose primary objective is to identify the dose(s) of Hycofenix oral solution that results in exposures of hydrocodone bitartrate, pseudoephedrine hydrochloride, and guaifenesin in children (6 to 11 y
Enrollment scheduled to begin September 2016.
Hycofenix® (Hydrocodone, Pseudoephedrine, and Guaifenesin)
Conduct an open-label, multi-dose safety and tolerability study in children (aged 6 to 11) and adolescents (aged 12 to 17 years). The population eligible for the study would be children and adolescents with cough/cold symptoms for whom a combin
Conduct a juvenile rat toxicology study to identify the unexpected serious risk of adverse effects of sumatriptan/naproxen on postnatal growth and development. The study should utilize animals of an age range and stage(s) of development that ar
Treximet® (Sumatriptan and Naproxen Sodium)
Conduct a pharmacokinetics (PK) study in children ages 6 years to 11 years with migraine. Using information from this PK study, conduct a controlled efficacy study in children ages 6 years to 11 years with migraine. Conduct a long-term open-lab
A controlled trial to evaluate the longer-term (i.e., maintenance) efficacy of aripiprazole in the treatment of pediatric patients (6-17 years) Tourette’s Disorder. This trial must include a placebo group and more than one fixed dose and must
OTSUKA PHARMACEUTICAL DEVELOPMENT AND COMMERCIALIZATION INC
A controlled trial to evaluate the longer-term (i.e., maintenance) efficacy of lisdexamfetamine in the treatment of adults with binge eating disorder. This trial must be placebo-controlled, utilize a randomized withdrawal design, and include an
A single pediatric study in at least 60 patients (30 per arm) undergoing cataract surgery
There are 68 subjects currently enrolled in the study of the 60-planned subject.
Conduct a 2-AB UHPLC assay on lots of Herceptin®(trastuzumab) derived from the drug substance manufactured at the Roche Penzberg facility without a media depth filter and used to supply the HERA, BCIRG006, and/or ToGA trials, and that are repr
A dose-ranging study assessing the pharmacokinetics, safety, tolerability, and effectiveness of Varubi (rolapitant) in pediatric patients ages 0-17 years old
A study to evaluate the efficacy and safety of a single oral dose of Varubi (rolapitant) in pediatric patients ages 0-17 years old
In vivo drug interaction study with a sensitive substrate of CYP2D6 to study the duration of CYP2D6 inhibition beyond 7 days after a single dose administration of Varubi (rolapitant)
Conduct an in vivo drug-drug interaction study to measure the effect of strong CYP3A4 inducers (e.g. rifampin) on the exposure to pimavanserin. Depending on the results of the study, a maximum dose could be recommended when CYP3A4 inducers are
Conduct a study of Entereg (alvimopan) for the acceleration of gastrointestinal recovery in pediatric patients aged 0 months to 10 years undergoing partial bowel resection surgery with primary anastomosis. The study will measure population phar
Conduct a phase 4 observational study evaluating the efficacy and safety of Neulasta®(pegfilgrastim) in the setting of Hematopoietic Syndrome (HS) following acute radiation exposure.
Conduct a study of Entereg (alvimopan) for the acceleration of gastrointestinal recovery in pediatric patients aged 10 to 18 years undergoing partial bowel resection surgery with primary anastomosis. The study will measure the time to first tol
Conduct a randomized phase 3 clinical trial in classical Hodgkin lymphoma that verifies and isolates the clinical benefit of nivolumab for patients with classical Hodgkin lymphoma. The primary endpoint would be progression-free survival as dete
Characterize complications after allogeneic hematopoietic stem cell transplantation (HSCT) following nivolumab in at least 90 patients with classical Hodgkin lymphoma, of which at least 50% had received nivolumab alone or in combination as the
Conduct “GO29294: A Phase 3, Open-label, Multicenter, Randomized Study to Investigate the Efficacy and Safety of Atezolizumab Compared with Chemotherapy in Patients with Locally Advanced or Metastatic Urothelial Bladder Cancer After Failure w
Develop and validate an assay with improved sensitivity for the detection of neutralizing antibodies against atezolizumab in the presence of atezolizumab levels that are expected to be present in samples at the time of patient sampling. Patient
Conduct a clinical trial to evaluate the effect of atezolizumab on thyroid function tests and clinical thyroid disease. Submit the datasets with the completed report.
Submit the median duration of response for patients who responded to atezolizumab on GO29293. This includes all patients and patients whose tumorinfiltrating cells stain IC 2/3 or IC 0/1. Submit the datasets with the completed report.
Conduct an animal study that will measure the effect of PD-L1 inhibition on the magnitude of the primary (1st vaccination) and recall (2nd vaccination) antibody responses to antigen challenge (e.g., KLH). This study will evaluate the effect of
Conduct a study to analyze laboratory data including serum complement, IgE, tryptase, histamine, and human anti-chimeric antibody levels obtained in patients with documented Grade 4 allergic reactions or anaphylaxis from a sufficient number of
Develop and validate an assay with improved sensitivity for the detection of neutralizing antibodies against dinutuximab in the presence of dinutuximab levels that are expected to be present in samples at the time of patient sampling.
To conduct a study to assess the neutralizing anti-drug antibody responses to dinutuximab with a validated assay capable of sensitively detecting neutralizing antibody responses in the presence of dinutuximab levels that are expected to be pres
To conduct a 5-month repeat-dose juvenile animal toxicology study in cynomolgus monkeys that will measure the chronic toxicity of dinutuximab, particularly its effects on the central and peripheral nervous system. Administration of dinutuximab
Conduct a randomized, double blind, double-dummy, active- and placebo controlled, 4-period crossover dose-ranging study with vilanterol inhalation powder in children 5 to 11 years of age with asthma. Each treatment period will be of one-week du
Final protocol missed pending further discussion with Agency in 3Q2016
Breo® Ellipta® (Fluticasone Furoate and Vilanterol)
Conduct a 12 week randomized, double-blind, active controlled, safety and efficacy study with fluticasone furoate/vilanterol inhalation powder in children 5 to 11 years of age with asthma.
Conduct a 52 week randomized, double blind, active comparator, safety study with fluticasone furoate/vilanterol inhalation powder in children 5 to 11 years of age with asthma.
In vitro studies to evaluate the inhibitory potential of Varubi (rolapitant) on renal transporters, i.e., organic cation transporter 2 (OCT2), multidrug and toxin extrusion (MATE) transporters, organic anion transporter 1 (OAT1), and organic an
In vitro study to evaluate the inhibitory potential of Varubi (rolapitant) on OATP1B1 and OATP1B3. The in vitro study results will determine the need for a subsequent clinical assessment of a drug interaction between Varubi (rolapitant) and oth
Utilize the validated anti-adalimumab antibody (AAA) assay developed under PMR 2517-3 (as described in the FDA Fulfillment of Postmarketing Requirement Letter dated April 1, 2015) to analyze the immunogenicity profile of adalimumab using banked
Conduct an Enhanced Pharmacovigilance Study to evaluate the risk factors for and outcomes of severe visual loss following exposure to XALKORI (crizotinib). This study will include a mechanism to collect, classify, and analyze data on Grade 4 se
A single-dose, open-label, randomized pharmacokinetic study of Dyanavel XR (amphetamine extended release) oral suspension in male and female children (4 to less than 6 years of age) with ADHD in fed condition.
A randomized, double-blind, placebo-controlled, flexible-dose titration study of Dyanavel XR (amphetamine extended-release) oral suspension in children ages 4 to 5 years diagnosed with ADHD.
Deferral Extension Requested 09/30/2016. Denied per FDA letter dated 11/14/2016.
A one year Pediatric Open-Label Safety Study of patients age 4 to 5 years (at the time of entry into PMR 2970-1 or PMR 2970-2, or at the time of enrollment if directly enrolled into PMR 2970-3) diagnosed with ADHD treated with Dyanavel XR (amph
Conduct a safety, pharmacokinetics, and hypothalamic-pituitary-adrenal (HPA) axis suppression study of Ultravate (halobetasol propionate) lotion, 0.05% under maximal use conditions in adolescents 12 years to 16 years 11 months of age with plaqu
Conduct a multicenter, evaluator-blinded, randomized comparator study designed to evaluate the safety, efficacy and pharmacokinetics of IV daptomycin administered for up to 14 days in the treatment of pediatric patients ages 1 to 17 years with
Conduct a trial to evaluate pediatric pharmacokinetics, safety and antiviral activity of dolutegravir in HIV-1 infected integrase strand transfer inhibitor-naïve, pediatric subjects weighing less than 15kg and at least 4 weeks in age. Initial
The Sponsor reports that the study is being conducted.
Conduct a trial to evaluate pediatric pharmacokinetics, safety and antiviral activity of dolutegravir in HIV-1 infected integrase strand transfer inhibitor-naïve, pediatric subjects weighing 15kg to less than 30kg. Initial evaluation of dolute
Conduct a trial to evaluate pediatric pharmacokinetics, safety and antiviral activity of dolutegravir in HIV-1 infected integrase strand transfer inhibitor-naïve, pediatric subjects less than 12 years of age weighing 30 kg to less than 40kg. I
Per FDA letter dated 09/14/2016, this PMR has been fulfilled.
Conduct a trial to evaluate pediatric pharmacokinetics safety and antiviral activity of dolutegravir in HIV-1 infected pediatric subjects weighing 20 to less than 40kg, who are integrase strand transfer inhibitor (INSTI) experienced with certai
Conduct a trial to evaluate pediatric pharmacokinetics, safety and antiviral activity of dolutegravir in HIV-1 infected pediatric subjects 12 years to less than 18 years of age and weighing at least 40kg, who are integrase strand transfer inhib
Conduct a repeat dose, dose ranging pharmacokinetic/pharmacodynamics (PK/PD) study evaluating Rayaldee (calcifediol) in predialysis patients with stage 3 or 4 chronic kidney disease with secondary hyperparathyroidism and 25-OH vitamin D levels
Conduct a 16-week, randomized (1:1), placebo-controlled, double-blind, efficacy and safety study evaluating Rayaldee (calcifediol) in predialysis patients with stage 3 or 4 chronic kidney disease with secondary hyperparathyroidism and 25-OH vit
A postmarketing study that will allow you to test for and control the levels of the following elemental impurities in your product: [....] present during the manufacturing process and have the potential to contaminate your product. As part of y
The supplements were approved. The PMR is delayed because the final study report date for all NDAs specified in this PMR were missed
SODIUM CHLORIDE 0.9% IN PLASTIC CONTAINER
Dextrose and Electrolyte® No 48 (Dextrose and Multiple Electrolytes Type 1)
Plasma-Lyte® 148 Injection in Plastic Container
Clinimix® Sulfite-Free (Amino Acids in Dextrose)
Plasma-Lyte® 56 in 5% Dextrose Injection in Plastic Container
Calcium Chloride and Magnesium Chloride in Dextrose
SODIUM CHLORIDE 3% & 5% INJ USP (PL 146)
LACTATED RINGER'S INJ IN PLASTIC(PL-146)
SODIUM CHLORIDE 0.45% IN PLASTIC CONTAIN
Dextrose, Electrolytes, and Potassium Chloride
CLINIMIX E 2.75/10 SULFITE-FREE W/ ELECT
Conduct a prospective observational study utilizing data from the United Network for Organ Sharing (UNOS) on the incidence rates of post-transplant lymphoproliferative disorder (PTLD) in US adult kidney-only transplant recipients who are treate
Provide evidence sufficient to characterize the long-term safety of ruxolitinib in the treatment of patients with polycythemia vera with splenomegaly from trials CINC424B2301 and INCB18424-256. Submit the complete final reports and data showing
Conduct a trial to provide evidence sufficient to characterize the long-term safety of ruxolitinib in the treatment of patients with polycythemia vera who do not have splenomegaly and who have had an inadequate response to or are intolerant of
Establish reliability requirements for the combination product Narcan Nasal Spray (naloxone hydrochloride), and complete testing which verifies the combination product reliability.
Establish procedures for monitoring reports of failure of the combination product Narcan Nasal Spray (naloxone hydrochloride) to activate or failure of the combination product to deliver the full-labeled dose. Provide interim and final reports
Conduct an adequate leachable safety assessment for the [....] used in your container closure system. This assessment must include leachable data from long -term stability studies testing at least three batches (taking into consideration the pr
Conduct a long-term stability evaluation placing at least three (3) manufactured lots of NARCAN Nasal Spray, 40 mg/mL, on long-term stability evaluation at the following temperatures: a. 2 to 8°C b. 40°C/75% RH - to extend the time points out
Conduct an elotuzumab exposure-response analysis for efficacy and safety utilizing data from trial CA204006. The result of the exposure-response analyses from both CA204004 and CA204006 will be used to determine whether a postmarketing trial is
Twenty-eight day, daily, repeat dose, oral gavage dose-range finding toxicity study in neonatal rats to provide rationale for dose selection for the three-month neonatal rat toxicity study with Syndros (dronabinol oral solution).
A repeat dose, randomized, active-controlled trial to assess the safety of Sustol (granisetron) extended-release injection, for subcutaneous use, when administered to patients receiving chemotherapy, for 6 cycles.
A 2-year subcutaneous carcinogenicity study in rats with the [...] vehicle used in the formulation of Sustol (granisetron) extended-release injection, for subcutaneous use.
A Syrian Hamster Embryo (SHE) cell transformation assay with the [...] vehicle used in the formulation of Sustol (granisetron) extended-release injection, for subcutaneous use.
An addition to the stability protocol to test the break force, glide force, and peak force of injection of Sustol (granisetron) extended-release injection, for subcutaneous use.
Develop and validate an improved assay for detecting Galsulfase in human plasma.
This PMC final report was due on 3/31/2006. FDA issued a not fulfilled letter on 4/28/2011. The sponsor is working with the FDA to discuss options to fulfill the PMC.
Randomized controlled trial in pediatric patients, including neonates, comparing Liposyn II 20% IV Fat Emulsion with a phytosterol-depleted formulation of Liposyn II 20% IV Fat Emulsion to evaluate the incidence of liver injury, including eithe
A study to assess the pharmacokinetics of ProAir RespiClick in pediatric asthma patients between the ages 4 to 11 years.
Per FDA letter dated 04/28/2016, this PMR has been fulfilled.
A study to assess the efficacy and safety of two dose levels of ProAir RespiClick in pediatric asthma patients between the ages 4 to 11 years.
A study to assess the chronic dose efficacy and safety of ProAir RespiClick in pediatric asthma patients between the ages 4 to 11 years.
Establish a registry for children aged 2 to < 6 years to enroll approximately 200 patients receiving deferasirox and follow them for 5 years. Collect data at least monthly for renal function and blood pressure and yearly for growth and developm
Conduct a trial to assess the long-term efficacy and safety of deferasirox in patients with NTDT and high LIC. The trial should assess response rates in the subset of patients with baseline LIC values >15 mg Fe/g dw (proportion of patients achi
Conduct a trial to assess the long-term efficacy (and safety) of deferasirox treatment to a target LIC of 3 mg Fe/g dw followed by one or more treatment holidays until the LIC is ¡Ý5 mg Fe/g dw in patients with NTDT. Follow-up of all subjects
Conduct a prospective, randomized trial in at least 210 patients with low to intermediate risk myelodysplastic syndromes (MDS) receiving deferasirox for transfusional iron overload (approximately 140 patients) or placebo (approximately 70 patie
Conduct a study, using your established registry, to evaluate the risk of growth inhibition in children (aged 10 to <18 years old at enrollment) with NTDT and treated with deferasirox for documented iron overload. Follow at least 40 children fo
Conduct an enhanced pharmacovigilance study, including proactive surveillance and follow-up of spontaneous reports, to characterize the frequency and severity of adverse Events of Special Interest (ESIs), defined as all deaths and severe or ser
Complete a study of long-term follow-up (3 years) in 150 patients with myelodysplastic syndromes (MDS) receiving deferasirox to evaluate safety (including cardiac, hepatic, endocrine and renal) and hematologic and clinical benefit of deferasiro
Conduct a trial to assess ocular toxicity in patients receiving deferasirox. Examinations should include distance visual acuity, applanation tonometry, lens photography, and wide angle fundus photography of retina and optic nerve and should be
Conduct a trial to assess the long-term safety of deferasirox in patients with NTDT by conducting a trial of deferasirox for the treatment of iron overload (LIC ¡Ý5 mg Fe/g dw) in non-transfusion dependent thalassemia (NTDT) in patients aged
An open-label, 26-week, randomized, controlled efficacy and safety trial comparing Tresiba (insulin degludec injection) with insulin detemir in pediatric patients with type 1 diabetes ages 1 to 17 years (inclusive) using insulin aspart at each
Conduct a randomized, double-blind, active-controlled trial evaluating the effect of Tresiba (insulin degludec injection) on the incidence of major adverse cardiovascular events (MACE) in subjects with type 2 diabetes mellitus. The primary obje
To develop and validate an assay to assess for the presence of anti-degludec antibodies that has a sensitivity consistent with FDA guidance. Your final report should include a summary of the validation exercise including supporting data, a summ
To assess the incidence and titers of anti-degludec antibodies in sera from patients treated with Tresiba (insulin degludec injection) in Tresiba (insulin degludec injection) clinical trials and determine whether they are associated with differ
An open-label, 16-week, randomized, controlled efficacy and safety trial comparing Ryzodeg 70/30 (insulin degludec and insulin aspart injection) administered once daily with a main meal and insulin aspart for additional meals to insulin detemir
Ryzodeg® 70/30 (Insulin Degludec and Insulin Aspart)
To assess the incidence and titers of anti-degludec antibodies in sera from patients treated with Ryzodeg 70/30 (insulin degludec and insulin aspart injection) in Ryzodeg 70/30 (insulin degludec and insulin aspart injection) clinical trials and
In vitro drug-drug interaction (DDI) studies in media mimicking physiological conditions (i.e., at pH 1.2, 4.5 and 6.8), including appropriate replicates. The DDI studies may be conducted in the absence of potassium, thus representing a “wors
Conduct a 24-week, randomized, controlled efficacy and safety study comparing Adlyxin (lixisenatide) with placebo in patients with type 2 diabetes ages 10 to 17 years (inclusive), followed by a 28-week double-blind controlled extension. Subject
Deferred pediatric study under PREA for the treatment of HIV-1 infection in pediatric subjects from 2 years to 6 years of age. This study will determine the pharmacokinetic profile, safety, and activity of etravirine in pediatric subjects from
Azelaic acid pre-foam emulsion: 104-week dermal carcinogenicity study in CD-1 mice
Conduct a safety and efficacy study of lubiprostone in pediatric patients with irritable bowel syndrome with constipation (IBS-C) ages 6 years to < 18 years. The design will consist of a 12-week multi-center, double-blinded, placebocontrolled s
Conduct an open-label extension safety study of lubiprostone, including a safety evaluation of the effects of lubiprostone treatment on bone growth, in pediatric patients ages 6 years to < 18 years with irritable bowel syndrome with constipatio
Conduct an in vitro study to characterize and quantify the degradants of the immediate release omeprazole of Yosprala at various pHs (i.e., pH 1, 1.5, 2, 2.5, 3, 3.5, 4) following a minimum of 1 hour of exposure at 37°C, and evaluate the diffe
Conduct a clinical PK trial evaluating the systemic exposures of the omeprazole degradants that are shown to be present at a higher level at pH <3.0 compared to higher pHs in the in vitro studies (PMC #3111-1). This trial will include both Yosp
A 6-week randomized, double blind, placebo-controlled trial in children 5 to 17 years of age with active, mild to moderate distal ulcerative colitis (extending up to 40 cm from the anal verge). The trial will evaluate pharmacokinetics (PK), eff
The final protocol milestone of this post-marketing commitment was missed due to challenges developing an appropriate trial for the pediatric population.
Provide nationally representative drug utilization data to FDA of sufficient detail that use of Truvada® for a pre-exposure prophylaxis (PrEP) indication and individuals using Truvada® for a PrEP indication can both be characterized. These da
An Enhanced Pharmacovigilance Study to evaluate the risks factors and clinical sequelae of immune-mediated encephalitis following exposure to OPDIVO (nivolumab). This study will include a mechanism to collect, classify, and analyze data on mode
Trial1321.3 titled, “A Phase III, Case Series Clinical Study of the Reversal of the Anticoagulant Effects of Dabigatran by Intravenous Administration of 5.0 g Idarucizumab in Patients Treated with Dabigatran Etexilate Who Have Uncontrolled Bl
Conduct a prospective, long-term, observational study in STRENSIQ (asfotase alfa) treated patients with perinatal/infantile-onset and juvenile-onset hypophosphatasia (HPP) from ages birth and older. The purpose of the study is to assess the lon
Develop an assay to directly compare the complement activating capacity of STRENSIQ (asfotase) alfa to that of human IgG1. The assay should be set up under conditions to readily detect complement activation by IgG1. A dose response curve to dem
Develop a validated cross-reactive immunologic material (CRIM) assay for patients with hypophosphatasia (HPP) and test patient samples in a cohort of untreated patients. Results should be correlated with antibody response (binding and neutraliz
Conduct a safety and efficacy study in pediatric patients with chronic idiopathic constipation ages 2 to 5 years treated with Linzess (linaclotide).
Conduct a safety and efficacy study in pediatric patients with chronic idiopathic constipation ages 6 to 17 years treated with Linzess (linaclotide).
105 patients out of the planned 120 patients have been enrolled into the study..
A randomized, double-blind, placebo-controlled, flexible-dose titration study of methylphenidate hydrochloride extended-release capsules (Aptensio XR) in children ages 4 to 5 years diagnosed with ADHD
A single-dose, open-label, randomized pharmacokinetic study of Aptensio XR capsules in male or female children (4 to less than 6 years of age) with ADHD in fed condition.
A one year Pediatric Open-Label Safety Study for patients age 4 to 5 years (at the time of entry into Study 1 or Study 2 or at the time of enrollment if directly enrolled into Study 3) with ADHD.
Perform immunogenicity testing on anti-drug antibody (ADA)-positive samples from clinical studies of type 2 diabetes patients treated with lixisenatide to determine the incidence of neutralizing antibodies (NAb) and anti-lixisenatide antibodies
Conduct and submit the results of at least one multicenter, randomized clinical trial establishing the superiority of pembrolizumab over available therapy as determined by an improvement in overall survival in patients with metastatic squamous
Conduct a pharmacokinetic and safety study of an age-appropriate formulation of Troxyca ER (oxycodone and naltrexone) in patients seven to less than 17 years of age with pain severe enough to require daily, around-the-clock, long-term opioid tr
In order to provide the baseline data to support the hypothesis-testing studies required under PMR 2965-3, conduct a descriptive study that analyzes data on the following: (1) utilization of TROXYCA ER (oxycodone and naltrexone) and selected co
Conduct formal observational studies to assess whether the properties intended to deter misuse and abuse of TROXYCA ER (oxycodone and naltrexone) actually result in a meaningful decrease in misuse and abuse, and their related clinical outcomes,
Conduct and submit the results of at least one multicenter, randomized clinical trial establishing the superiority of alectinib over available therapy in patients with metastatic anaplastic lymphoma kinase positive non-small cell lung cancer (N
Complete a pharmacokinetic trial to determine an appropriate dose of alectinib in patients with moderate to severe hepatic impairment in accordance with the FDA Guidance for Industry entitled “Pharmacokinetics in Patients with Impaired Hepati
Enhanced Pharmacovigilance Study to Assess and Analyze the Risks of Hypotension, Syncope, Accidents or Injuries, and Fatal Outcomes with Use of Addyi (flibanserin).
Prospective Observational Study to Evaluate the Risk of Appendicitis with the Use of Addyi (flibanserin) in Women Aged 18 to 44.
Pregnancy Registry Study to Evaluate Adverse Pregnancy Outcomes and Birth Defects in Pregnancies Exposed to Addyi (flibanserin).
Maternal-Fetal Outcome Study to Evaluate Adverse Pregnancy Outcomes and Birth Defects in Pregnancies Exposed to Addyi (flibanserin).
Alcohol Interaction Trial in the Target Female Population (Between Ages 18 and 44) to Evaluate the Interaction Between Addyi (flibanserin) and Alcohol Through a “Worst Case Scenario” with Varying Quantities of Alcohol Intake.
Alcohol Interaction Trial in the Target Female Population (Between Ages 18 and 44) to Evaluate the Interaction of the Timing of Alcohol Intake Relative to Addyi (flibanserin) Dosing. Dose(s) to be evaluated in this trial will be selected based
Alcohol Interaction Trial in the Target Female Population (Between Ages 18 and 44) to Evaluate the Interaction Between Addyi (flibanserin) and Alcohol Intake in a “Typical Real World Use” Setting. Dose(s) to be evaluated in this trial will
Conduct an open-label study to evaluate the pharmacokinetics and safety of BELBUCA in patients 7 through 16 years with pain severe enough to require daily, around-the-clock, long-term opioid treatment, and for which alternative treatment option
Conduct a multiple ascending dose clinical trial in adults to determine the maximum tolerated dose of BELBUCA without co-administration of naltrexone to inform the dosing for a thorough QT (tQT) trial of BELBUCA.
Conduct a thorough QT trial in adults without naltrexone co-administration to assess the risk of QT prolongation with BELBUCA. This trial will provide information on the conduction effects of BELBUCA on the heart, specifically cardiac repolariz
To submit a final report that includes updated results for overall survival for trial MO18264 (PRIMA), "A Multicenter, Phase III, Open-Label, Randomized Study in Patients with Advanced Follicular Lymphoma Evaluating the Benefit of Maintenance T
The final report was originally due Dec 31, 2013. On November 7, 2013, the milestones were revised to submit final follow-up data December 2017. The Sponsor is extending the follow-up period for the trial to obtain more survival information for
Deferred pediatric studies under PREA for the reduction of blood pressure in pediatric patients ages 1 to 16 years old.
Enrollment in the adolescent cohort (Age 12 through less than <18 years old) was completed on 30 September 2014 and was evaluated by the study DSMB on 10 November 2014. Enrollment of subsequent age cohorts has not proceeded, as agreed between t
Conduct a controlled postmarketing study to track adverse events in the recipients of Trima Accel collected platelets stored in 65% Isoplate/ 35% plasma. Adverse events (e.g., transfusion reactions) will be captured through active surveillance
Conduct a study to evaluate the immunogenic potential of the terminal heat-treated product.
Alphanate, Antihemophilic Factor/von Willebrand Factor Complex (Human)
Conduct a Postmarketing study to further evaluate safety and efficacy of the product in type 3 vWD patients undergoing major surgeries.
Conduct a post-marketing observational study to compare the safety profile of Vivotif® lots with release titers in the range of 6.9 x 109 to 10 x 109 CFU/capsule to lots with release titers in the range of 4 x 109 to 6 x 109 CFU/capsule.
Vivotif, Typhoid Vaccine Live Oral Ty21a
Conduct two clinical trials: Trial 1: A pilot study of the safety and pharmacokinetics of Prolastin-C administered weekly at a dose of 120 mg/kg. Trial 2: An adequately-powered study of clinically meaningful endpoints -Prolastin-C efficacy stud
Prolastin; Prolastin-C, Alpha-1-Proteinase Inhibitor (Human)
Conduct a study to evaluate T.R.U.E. TEST and the seven new allergens in pediatric patients ages six to seventeen.
Deferral Extension Requested December 16, 2013. Deferral Extension Granted per FDA letter dated December 30, 2013. A second Deferral Extension Requested November 10, 2015. Deferral Extension Denied per FDA letter dated December 21, 2015. The fi
T.R.U.E. TEST, Thin-Layer Rapid Use Epicutaneous Patch Test
Final Protocol Submission: November 30, 2016 Study/Trial Completion: To be determined should an event occur. Final Report Submission: To be determined should an event occur.
Emergent BioDefense Operations Lansing LLC
Submit results from your clinical studies using this product in populations for which your seasonal influenza vaccine is already licensed.
Establish a pregnancy registry that will enroll women exposed to Fluzone Quadrivalent during pregnancy and collect data on their outcomes and newborn health status.
Fluzone; Fluzone High Dose; Fluzone Intradermal; Fluzone Quadrivalent, Influenza Virus Vaccine
To establish a pregnancy registry that will enroll women exposed to Fluzone Intradermal Quadrivalent during pregnancy and collect data on their outcomes and newborn health status. Annual reports for this registry will be submitted with the Peri
Conduct an observational postmarketing safety surveillance study of FluMist Quadrivalent in children 2 years through 8 years of age.
FluMist; FluMist Quad, Influenza Vaccine Live, Intranasal
Conduct an observational postmarketing case-control study of the effectiveness of FluMist Quadrivalent in children 2 years through 17 years of age.
Conduct and report the results of a pilot trial to determine the effect of regular administration of the product on one or more clinically meaningful endpoint(s). Examples of acceptable endpoints include pulmonary exacerbations, serial pulmonar
Aralast; Aralast NP, Alpha-1-Proteinase Inhibitor (Human)
Contingent on the outcome of the pilot trial described in commitment 3, conduct and report the results of an adequately-powered study of clinically meaningful endpoints(s). Based on the results of the pilot study and the available scientific da
Conduct a non-interventional 2-armed study to evaluate the safety of Octagam Immune Globulin Intravenous (Human) 5% liquid preparation designed to monitor, analyze and report thrombotic events.
“Good Cause justification was submitted on 06/10/2015 (STN 125062/450). Good Cause was granted per FDA letter dated 07/22/2015. The study completion date has passed and the study has not yet been completed.”
OCTAGAM, Immune Globulin Intravenous (Human)
Conduct a randomized study of the product in surgery, comparing the safety and efficacy of the product administered by intermittent bolus infusion versus continuous infusion.
The trial was initiated in April 2006; however, due to slow subject recruitment, Baxter sought guidance from the FDA in 2010 to revise the study protocol and complete the study in a timely manner. Based on discussions with FDA the protocol was
Advate, Antihemophilic Factor (Recombinant), Plasma/Albumin Free Method
Conduct a pilot trial of clinically meaningful endpoint(s). Examples of acceptable endpoints include pulmonary exacerbations, serial pulmonary functions, and serial quantitative computerized axial tomographic (CT) lung scans.
Based on the results of the pilot study and the available scientific data at the time that this study is being designed, ZLB Behring will work with entities maintaining registries of alpha1-proteinase inhibitor deficient patients and with the N
Conduct a Phase II open-label administration (laboratory personnel will be blinded to group assignment) parallel-group, multicenter study to evaluate the safety and immunogenicity of two doses of Menactra when administered alone at 9 months and
Menactra, Meningococcal Groups (A, C, Y, and W-135) Polysaccharide Diphtheria Toxoid Conjugate Vaccine
Conduct a Phase IV, descriptive, epidemiological, safety surveillance study in the United States, entitled, Post-licensure Safety Surveillance Study of Menactra® Vaccine When Administered as a 2-dose Schedule to Children 9 Months through 23 Mo
Work with the FDA, and other public agencies as appropriate, to design and implement a clinical study protocol to include VIGIV dose ranging, and conduct a study to describe the treatment and clinical course of patients receiving VIGIV for comp
CNJ-016, Vaccinia Immune Globulin Intravenous (Human)
Conduct a large scale observational study of US subjects 50 to 59 years of age vaccinated with ZOSTAVAX to assess the long-term effectiveness of the vaccine and better characterize the duration of protection against herpes zoster (HZ).
Collaborate with the cancer registries in four countries in the Nordic Region (Sweden, Norway, Iceland, and Denmark) to assess long-term outcomes following administration of GARDASIL.
GARDASIL, Human Papillomavirus Quadrivalent (Types 6, 11, 16 and 18) Vaccine, Recombinant
Conduct a study in collaboration with the Norwegian Government, if GARDASIL is approved in the European Union and the Government of Norway incorporates HPV vaccination into its national guidelines, to assess the impact of HPV vaccination on the
Provide data concerning duration of immunity following administration of GARDASIL from the Nordic Long-Term Follow-up Study.
Conduct an observational safety study in a U.S. Managed Care Organization (MCO) to assess the general short-term safety of the vaccine as it is administered to a larger male population 9 through 26 years of age.
Conduct a long-term evaluation of vaccine effectiveness in males by extending Protocol 020. Study subjects will be follow-up for up to 10 years from day of enrollment into the base study (i.e., Day 1) for the occurrence of external genital lesi
Conduct ongoing study D-QIV-004 under PREA for active immunization for the prevention of disease caused by influenza A subtype viruses and type B viruses contained in Fluarix® Quadrivalent, in pediatric patients ages 6 months to 35 months of a
FLUARIX PMR Final Study Report to fulfill PMR #1 under /513 (STN 125127/833) submitted on 19-Jan-2017.
FLUARIX; FLUARIX Quadrivalent, Influenza Virus Vaccine
To establish a pregnancy registry to prospectively collect data on spontaneously-reported exposures to Fluarix® Quadrivalent during pregnancy.
A descriptive study of the safety of Pentacel with regard to selected medically attended events in at least 10,000 infants who will be followed from the first dose of Pentacel through approximately 6 months after the fourth dose.
Pentacel, Haemophilus b Conjugate Vaccine (Tetanus Toxoid Conjugate) Reconstituted with Diphtheria and Tetanus Toxoids and Acellular Pertussis Vaccine Adsorbed Combined with Poliovirus Vaccine Inactivated
Conduct a prospective cohort study in the military population receiving ACAM2000 to determine general safety information on expected and unexpected adverse events; Additional information on the natural history and clinical outcome of myocarditi
ACAM2000, Smallpox (Vaccinia) Vaccine, Live
Implement an enhanced surveillance program to ascertain at least 75% of symptomatic cases of myocarditis and pericarditis after ACAM2000 vaccination.
Implement a myocarditis registry with cases identified from routine surveillance to further understand the natural history of myocarditis and pericarditis after ACAM2000 vaccination, and to evaluate potential risk factors associated with the de
Deferred pediatric study under PREA to evaluate the safety and immunogenicity of FluLaval Quadrivalent in childeren 6 to 35 months of age.
STN 125163/405 fulfills postmarketing requirement #1 identified in the August 15, 2013 approval letter for STN 125163/253.
FluLaval; FluLaval Quadrivalent, Influenza Vaccine
Establish a pregnancy registry that will enroll women exposed to FluLaval® Quadrivalent during pregnancy and collect data on their outcomes and newborn health status.
Deferred pediatric study under PREA to evaluate the safety and immunogenicity of FluLaval Quadrivalent in children 6 to 35 months of age.
STN 125163/405 fulfills postmarketing requirement #1 identified in the August 16, 2013 approval letter for STN 125163/254.
Conduct a study, based on a registry design, to assess the risk of cerebrovascular events in 1,500 patients with prostate cancer who receive sipuleucel-T.
Good cause granted per FDA letter dated April 6, 2016. Study completion date has passed and the study has not yet been completed. Delayed with good cause determination. Due date was extended: Study Completion: January 31, 2017 Final Report Subm
Conduct a study to investigate the cause of hemolysis, especially the specificity of the antibody involved. The study will enroll ITP and/or PID patients.
Privigen, Immune Globulin Intravenous (Human), 10% Liquid
Initiate a Registry to capture data for any patient who is prescribed CEPROTIN, irrespective of diagnosis.
Establish a pregnancy registry to prospectively collect data on spontaneously-reported exposures to Afluria during pregnancy.
AFLURIA; Afluria Quadrivalent, Influenza Vaccine
Deferred pediatric study under PREA, to evaluate the immunogenicity and safety of Afluria Quadrivalent in the pediatric population 5 through 17 years of age.
Supplement STN 125254/642 submitted on October 31, 2016 to fulfill PMR#1 approved under STN 125254/565.
Deferred pediatric study under PREA, to evaluate the immunogenicity and safety of Afluria Quadrivalent in the pediatric population 6 months through 4 years of age.
To establish a pregnancy registry to prospectively collect data on reported exposures to Afluria Quadrivalent during pregnancy and evaluate pregnancy outcomes. The registry will enroll a minimum of 500 evaluable subjects
Conduct ongoing long term efficacy study for HPV-015.
Cervarix, Human Papillomavirus Bivalent (Types 16 and 18) Vaccine, Recombinant
Conduct ongoing long term efficacy study for HPV-040.
Conduct an observational database study using the CPRD GOLD to evaluate the risk of neuroinflammatory/ophthalmic new onset of autoimmune disease(s) (NOAD) and other pre-specified NOAD within 12 months following the administration of the first d
Conduct a randomized trial using two regimens for prophylaxis.
XYNTHA; XYNTHA SOLOFUSE, Antihemophilic Factor (Recombinant), Plasma/Albumin Free
Conduct a large-scale observational post-licensure safety study in the U.S to assess the potential serious risk of intussusception and other serious adverse effects (specifically Kawasaki disease, hospitalizations due to acute lower respiratory
Conduct a clinical trial designed to evaluate higher than labeled dose schedules of CINRYZE for routine prophylaxis of angioedema attacks in patients with Hereditary Angioedema (HAE).
Conduct study DoD Active Surveillance in 20,000 patients.
Ixiaro, Japanese Encephalitis Vaccine, Inactivated, Adsorbed
Conduct the DoD Pregnancy Surveillance study.
Conduct study IC51-325 " Long Term Immunogenicity And Safety With Or Without A Booster Dose Following Primary Vaccination With The Japanese Encephalitis Vaccine IC51 (IXIARO®) In A Pediatric Population In JEV - Endemic Countries." An open-labe
The Final Study Report was submitted to FDA on June 30, 2015. Since this is a PREA PMR, the Final Study Report does not fulfill the PREA requirement.
Conduct post-marketing assessments of human factors issues that may affect preparation and administration of the 0.25 mL dose.
Evaluate immunogenicity in patients with repeat exposure to ATryn via an immunosurveillance program to detect antibodies to ATryn, endogenous Antithrombin, goat Antithrombin and goat milk proteins.
Conduct a reactogenicity and immunogenicity study under PREA for active immunization for the prevention of disease caused by influenza virus subtypes A and type B contained in Flublok, in children ages 6 years through 17 years.
Per FDA letter dated 02-Feb-2016, this PMR has been released.
Conduct a reactogenicity and immunogenicity study under PREA for active immunization for the prevention of disease caused by influenza virus subtypes A and type B contained in Flublok, in children ages 3 years through 5 years.
Per FDA letter dated October 7, 2016, this PMR has been released.
Establish a pregnancy registry to collect data prospectively on an actively recruited cohort of 600 pregnant women, of whom at least 300 will have been exposed to Flublok. The statistical analysis will include both exposed women and concurrentl
To conduct a confirmatory clinical efficacy and safety study (PSC12) in adults 50 years of age and older for active immunization for the prevention of disease caused by influenza virus subtypes A and types B contained in your investigational qu
To conduct an observational postmarketing safety study (PSC13) in approximately 25,000 Flublok recipients 18 years of age and older to further characterize the safety profile of Flublok using recipients of U.S.-licensed, egg-based, trivalent or
Deferred pediatric study (Study PSC08) under PREA to evaluate the safety and immunogenicity of a Flublok quadrivalent formulation in children ages 6 years through 17 years.
Per FDA letter dated 02-Feb-2016, this PMR has been fulfilled.
Deferred pediatric study (PSC17) under PREA to evaluate the safety and effectiveness of a Flublok quadrivalent formulation in children ages 6 years through 17 years.
Deferred pediatric safety, immunogenicity and efficacy study (PSC17) in children 3 years through 17 years of age for active immunization for the prevention of disease caused by influenza virus subtypes A and types B contained in Flublok Quadriv
Conduct a study to assess inhibitory antibody formation against C1-Esterase Inhibitor.
To evaluate the safety of the product, establish and maintain a registry for 3 years of patients treated with Berinert for any indication.
Conduct a randomized masked trial that would be adequately powered to more precisely define the efficacy of Berinert for acute facial HAE attacks.
Conduct a postmarketing surveillance study, your "Sentinel Surveillance Plan", to detect potential safety signals and to monitor and analyze uncommon and unexpected medical events occurring within 42 days following vaccination in the first 100,
Adenovirus Type 4 and Type 7 Vaccine, Live, Oral
Conduct Study No. V71_18, a randomized, observer-blind, non-inferiority immunogenicity and safety study with AGRIFLU and a US licensed trivalent inactivated Influenza Vaccine in a pediatric population from 3 years to 17 years of age.
The company submitted the Final Study report on Feb 28, 2013 under 125297/46. Since this is a PREA PMR, the Final Study Report does not fulfill the PREA requirement.
Conduct a randomized, observer-blind, immunogenicity and safety study with AGRIFLU and a US licensed trivalent inactivated Influenza Vaccine in a pediatric population from 6 months to less than 3 years of age.
Final Study Report was submitted as 125297/49 on July 31, 2013. Since this is a PREA PMR, the Final Study Report does not fulfill the PREA requirement.
Establish a pregnancy registry to prospectively collect data on spontaneously-reported exposures to AGRIFLU during pregnancy.
The study completion (establishment of the pregnancy registry) date has passed and the study has not yet been completed.
Conduct a non-inferiority immunogenicity study with AGRIFLU and a US-licensed trivalent inactivated seasonal influenza vaccine in a population of adults 50 years of age and older.
Conduct a randomized, comparative trial, designed primarily to evaluate the potential for immune interference of concomitant use of MENVEO with Human Papillomavirus (Types 6, 11, 16, 18) Recombinant Vaccine and a Tetanus Toxoid, Reduced Diphthe
Submission of the complete final study report is still delayed for this agreed upon PMC due to issues with the testing of the samples in the study for the HPV vaccine. Completed study report expected December 2017.
Menveo, Meningococcal (Groups A, C, Y, and W-135) Oligosaccharide Diphtheria CRM197 Conjugate Vaccine
Conduct a Phase IV self-controlled case-series study to assess the safety of Meningococcal (Groups A, C, Y, and W-135) Oligosaccharide Diphtheria CRM197 Conjugate Vaccine administered to a minimum of 50,000 HMO subjects 11 through 19 years of a
To conduct an open label, descriptive, epidemiological safety surveillance study of MENVEO vaccine in subjects 2 through 10 years of age. The study will include two parts. Part I of the study will begin with the first administration of MENVEO v
To conduct an open label, descriptive, epidemiological safety surveillance study of MENVEO in children 2 months through 23 months of age. The study will include two parts. Part I of the study will begin with the first administration of MENVEO t
Establish a U.S. pregnancy registry to collect safety data on spontaneously-reported exposures to Meningococcal (Groups A, C, Y, and W-135) Oligosaccharide Diphtheria CRM197 Conjugate Vaccine during pregnancy and lactation.
Conduct a phase 4 study to verify the clinical benefit by comparing the hemostatic efficacy of RiaSTAP to historical control.
Evaluate efficacy and safety in the peri-operative period in patients with congenital fibrinogen deficiency.
Evaluate efficacy and safety for routine prophylaxis in patients with congenital fibrinogen deficiency.
CSL Behring will conduct phase 4 study BI 3023_4003 to verify the clinical benefit by comparing the hemostatic efficacy of RiaSTAP™ to historical control.
Conduct an observational database study (6096A1-4005) to monitor the impact of Prevnar 13 in preventing overall invasive pneumococcal disease in Northern California Kaiser Permanente members, by estimating the impact of Prevnar 13 routine use o
Prevnar 13, Pneumococcal 13-valent Conjugate Vaccine (Diphtheria CRM197 Protein)
Conduct an observational study (6096A1-4010) to evaluate the impact of Prevnar 13 routine use in reducing acute otitis media (AOM) and nasopharyngeal colonization in young children caused by the serotypes in the vaccine.
Conduct study 6096A1-4018, an ecologic study to assess national trends in health care visits for otitis media in children younger than five years of age. This study will utilize ambulatory health care visit data from annual national surveys con
The Final Study Report expected December 31, 2017. Delay due to delay in receiving data from CDC database and was found an acceptable delay from submission of March 2, 2016. Delayed. Study is Ongoing, but behind original schedule. Final Study R
Conduct a study to evaluate concomitant use of Prevnar 13 and a U.S. licensed inactivated influenza vaccine in subjects greater-than or equal to 50 years of age, pre-immunized with 23-valent pneumococcal polysaccharide vaccine (23vPS).
Conduct a study exploring potential adverse events associated with protein aggregates. This study will evaluate the safety of the product following multiple repeat exposures over a period of at least 6 months of regular weekly administration. I
Deferral Extension Granted per FDA letter dated 01 February 2016. The final protocol due date has passed with no final protocol received.
Conduct a study investigating Epithelial Lining Fluid of Alpha1-Antitrypsin Deficient Patients for Alpha1-PI and analytes levels following augmentation therapy with GLASSIA. The primary endpoint for this study will be both antigenic and functio
Conduct a postmarketing clinical program for GLASSIA Investigating the Safety and Efficacy of GLASSIA vs. Placebo and another (Higher) dose of GLASSIA I.V. by Weekly Administration in Alpha1-Antitrypsin Deficient Patients with emphysema and des
Kamada Ltd. commits to conduct and report the results of a Human Factors study, to evaluate the types of problems that patients/caregivers may encounter in self-infusing. This information will be used to revise, as needed, the training program
Conduct Study Hib-097, a comparative safety and immunogenicity clinical trial of primary and booster immunization with Hiberix relative to U.S. licensed control vaccines.
Hiberix, Haemophilus b Conjugate Vaccine (Tetanus Toxoid Conjugate)
Conduct a study, based on a registry design, to assess the risk of skin cancer in the area of azficel-T injections and the risk of immune-mediated hypersensitivity reactions in 2700 patients who receive azficel-T.
Conduct a Phase IIIb open-label administration (laboratory personnel will be blinded to treatment), parallel-group, controlled, multicenter study to evaluate concomitant administration of MenHibrix with rotavirus, 13-valent pneumococcal conjuga
MENHIBRIX, Meningococcal Groups C and Y and Haemophilus b Tetanus Toxoid Conjugate Vaccine
Conduct a Phase IV, Multicentre, Open-Label Study to Evaluate the Efficacy and Safety of BIVIGAM in Primary Immune Deficiency Disorders in Children and Adolescents ages 2 to 16.
Bivigam, Immune Globulin Intravenous (Human)
Conduct a Prospective, non-interventional, active-control, observational safety study to further assess the potential risk of hypotension, hepatic and renal impairment in Biotest-IGIV-treated patients with primary humoral immunodeficiency (PI).
Conduct a deferred pediatric study under PREA for use of EVARREST as an adjunct to hemostasis in patients 1 month to 17 years undergoing intra-abdominal, retroperitoneal, pelvic, and non-cardiac thoracic surgical procedures.
Per FDA letter dated October 7, 2016, this PMR/PMC has been released
Deferred pediatric study under PREA for use as an adjunct to hemostasis for control of bleeding during liver surgery in the pediatric study for ages 30 days to 18 years.
Deferred pediatric study under PREA for use with manual compression as an adjunct to hemostasis in pediatric patients aged 1 month to less than 18 years undergoing surgery, when control of bleeding by conventional surgical techniques (such as s
Conduct a Post Approval Safety Study (PASS) NN1841-3868: "Use of rFXIII in treatment of congenital FXIII deficiency, a prospective multi-center observational study."
TRETTEN, Coagulation Factor XIII A-Subunit (Recombinant)
Assess the safety and effectiveness of Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen for topical (non-submerged) application to a surgically created vascular wound bed in the treatment of mucogingival disorders in pediatr
GINTUIT, Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen
Deferred pediatric study under PREA for the treatment of primary immunodeficiency in pediatric patients 2-16 years of age.
HYQVIA, Immune Globulin Infusion (Human), 10% with Recombinant Human Hyaluronidase
Baxter commits to conducting an evaluation of long-term safety study of HYQVIA in 250 patients with PID including up to 50 patients developing antibodies. The duration of the study is 6 years and subjects who become antibody positive will be el
Baxter commits to establish and maintain a pregnancy registry to assess a) the course and outcome of the pregnancy, b) the development of the fetus/infant at birth, and c) the development of the infant for two years following birth. The duratio
Conduct a study (Study V58_31) under PREA to evaluate the safety of FLUCELVAX in children ages 4 years to less than 18 years.
Per FDA letter dated 23-May-2016, this PMR/PMC has been fulfilled.
Flucelvax; Flucelvax Quadrivalent, Influenza Vaccine
Deferred pediatric dose ranging, immunogenicity and safety study (Study V58P16) under PREA in children ages 6 months to less than 4 years.
PMR Final Study Report submitted 30-Nov-2016.
Deferred pediatric study (Study V58_35) under PREA to evaluate the safety and immunogenicity of FLUCELVAX in children ages 6 months to less than 4 years.
Per FDA letter dated May 23, 2016, this PMR/PMC has been released.
Establish a pregnancy registry to prospectively collect data on spontaneously-reported exposures to FLUCELVAX during pregnancy.
Conduct a randomized, controlled, observer-blind, immunogenicity and safety study in adults comparing three lots of FLUCELVAX produced under manufacturing process 1.1 at the Holly Springs facility.
To conduct a study to evaluate the efficacy, safety and immunogenicity of the quadrivalent formulation of your Influenza Vaccine compared to a non-influenza comparator vaccine in persons 4 years to less than 18 years of age.
Deferred pediatric study (Study V130_10) under PREA to evaluate the safety and immunogenicity of your quadrivalent formulation of Influenza Vaccine in pediatric subjects 6 months to less than 4 years of age.
To establish a pregnancy registry to prospectively collect data on spontaneously-reported exposures to Flucelvax® Quadrivalent or Flucelvax® during pregnancy and collect data on pregnancy outcomes. When the registry has enrolled a minimum of
Conduct an open-label, multicenter, clinical study to investigate safety, tolerability and efficacy of Octaplas in the management of pediatric patients less than 16 years old who require multiple plasma coagulation factors
Deferral Extension Requested 06/29/2016. Deferral extension Granted per FDA letter dated 08/10/2016.
OCTAPLAS, Pooled Plasma (Human), Solvent/Detergent Treated
Cnduct a non-interventional, open-label, multicenter, clinical study to investigate safety, tolerability and efficacy of Octaplas in the management of pediatric patients less than 16 years old who require therapeutic plasma exchange.
Conduct a non-interventional 2-arm study to evaluate the safety of Octaplas versus FFP in patients treated for thrombotic thrombocytopenic purpura (TTP) with special emphasis on monitoring the occurrence of thromboembolic events (TEEs).
Conduct a non-interventional 2-arm study to evaluate the safety of Octaplas versus FFP in patients undergoing orthotopic liver transplantation (LTX) with a special emphasis on hyperfibrinolysis.
Conduct study Q-Pan H5N1=AS03-021 under PREA to evaluate the safety and immunogenicity of Influenza A (H5N1) Virus Monovalent Vaccine, Adjuvanted when administered to healthy persons 6 months to less than 18 years of age.
Per FDA letter dated September 9, 2016, this PMR has been fulfilled.
Q-Pan, Influenza A (H5N1) Virus Monovalent Vaccine, Adjuvanted
Conduct study Q-Pan-023 under PREA to evaluate the safety and immunogenicity of Influenza A (H5N1) Virus Monovalent Vaccine, Adjuvanted when administered to healthy children 6 months to less than 36 months of age.
Per FDA letter dated September 9, 2016, this PMR has been released.
Conduct study Q-Pan-024 under PREA to evaluate the safety and immunogenicity of Influenza A (H5N1) Virus Monovalent Vaccine, Adjuvanted when administered to healthy persons 6 months to less than 18 years of age. Study Q-Pan-024 will be conducte
Conduct study Q-Pan-025 under PREA to evaluate the safety and immunogenicity of Influenza A (H5N1) Virus Monovalent Vaccine, Adjuvanted when administered to healthy infants less than 6 months of age.
Establish a pregnancy registry in the U.S. that is able to prospectively collect data on an actively recruited cohort to study the safety of Influenza A (H5N1) Virus Monovalent Vaccine, Adjuvanted during pregnancy. A concept protocol for this p
A retrospective observational cohort study to estimate the risk of thromboembolic events following Vitamin K Antagonist (VKA) reversal among patients treated with Kcentra or Plasma for urgent reversal of VKA therapy in the setting of acute majo
Kcentra, Prothrombin Complex Concentrate (Human)
Conduct a prospective, double-blind, randomized, multicenter study to assess the efficacy of standard and low doses of Kcentra in reversing effects of VKA in subjects with acute major bleeding.
Sponsor has not submitted an acceptable protocol.
Deferred pediatric study IB1001-02B under PREA for the treatment of hemophilia B in pediatric patients ages 0-12. Final Protocol Submission: Received on November 6, 2015
The final protocol has been submitted but no subjects have yet entered the trial.
IXINITY, Coagulation Factor IX (Recombinant)
Evaluate the safety and efficacy of ALPROLIX in study 9HB02PED in the ongoing open-label, multi-center, evaluation of safety, pharmacokinetics, and efficacy in the prevention and treatment of bleeding episodes in pediatric subjects (PTPs) with
ALPROLIX, Coagulation Factor IX (Recombinant), Fc Fusion Protein
Evaluate the safety and efficacy of ALPROLIX in study 9HB01EXT, an open-label, multi-center evaluation of the long-term safety and efficacy in the prevention and treatment of bleeding episodes in previously treated patients with Hemophilia B.
Evaluation of long-term efficacy and safety of RIXUBIS in 100 patients of all age groups with hemophilia B, of which at least 25 will be subjects naïve to RIXUBIS.
RIXUBIS, Coagulation Factor IX (Recombinant)
You agree to re-initiate the BAT patient registry in the event of a mass botulism exposure scenario. The registry will evaluate the safety and clinical benefit of Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine) used to treat adu
BAT, Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine)
Conduct a patient registry study for 3 years to evaluate safety following BAT administration in adult, pediatric and infant patients with a confirmed or suspected exposure to botulism
Conduct a pharmacokinetic (PK) study in pediatric patients to verify the pediatric dosing recommendations for BAT.
Evaluate the safety and efficacy of Novoeight in previously untreated patients with hemophilia A in Trial NN7008-3809, Safety and efficacy study in prevention and treatment of bleeds in pediatric previously untreated patients with Hemophilia A.
Evaluate the safety and efficacy of Novoeight during long-term treatment of patients with severe and moderately severe hemophilia A in Trial NN7008-3553, Non-interventional safety and efficacy study during long term treatment of severe and mode
Conduct a deferred pediatric study (Study 140224) under PREA for immunotherapy for the treatment of grass pollen-induced allergic rhinitis with or without conjunctivitis confirmed by positive skin test or in vitro testing for pollen-specific Ig
Deferral Extension Requested 05/12/2016. Deferral Extension Granted per FDA letter dated 7/15/2016. Study is ongoing.
ORALAIR, Sweet Vernal, Orchard, Perennial Rye, Timothy, and Kentucky Blue Grass Mixed Pollens Allergen Extract
To conduct an observational postmarketing study (Study 140225) to further describe the safety profile in approximately 6,000 patients 10 to 65 years of age receiving Sweet Vernal, Orchard, Perennial Rye, Timothy, and Kentucky Blue Grass Mixed P
Conduct a post-market claims-based study to further describe the safety profile of GRASTEK in marketed use in the United States. The study will enroll all new users of GRASTEK identified through claims data from a large US health insurance data
GRASTEK, Timothy Grass Pollen Allergen Extract
Conduct a post-market electronic medical record study to further describe the safety profile of GRASTEK in marketed use in the United States. The study will enroll all new users of GRASTEK identified through electronic medical records in a larg
Conduct study, protocol #P008, under PREA to evaluate both safety and efficacy of RAGWITEK as immunotherapy for the treatment of short ragweed pollen-induced allergic rhinitis, with or without conjunctivitis, confirmed by positive skin test or
RAGWITEK, Short Ragweed Pollen Allergen Extract
Conduct study, protocol #P009, under PREA to evaluate safety of RAGWITEK as immunotherapy for the treatment of short ragweed pollen-induced allergic rhinitis, with or without conjunctivitis, confirmed by positive skin test or in vitro testing f
A postmarket claims-based study to further describe the safety profile of RAGWITEK in marketed use in the United States. The study will enroll all new users of RAGWITEK identified through claims data from a large U.S. health insurance database
A postmarket electronic medical record study to further describe the safety profile of RAGWITEK in marketed use in the United States. The study will enroll all new users of RAGWITEK identified through electronic medical records in a large U.S.
To complete the data analysis for trial 8HA02PED, An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein, BIIB031, in the Prevention and Treatment of Bleeding Epi
ELOCTATE, Antihemophilic Factor (Recombinant), Fc Fusion protein
To complete ongoing trial 8HA01EXT, An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previo
To complete trial 99HA306, An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Se
Conduct a study consisting of establishment and maintenance of a registry of HAE patients 13 years of age and older who are prescribed RUCONEST without plasma-derived C1 esterase inhibitors to evaluate the incidence of adverse events and time t
RUCONEST, C1 Esterase Inhibitor (Recombinant)
A post-marketing registry study of perioperative management of moderate to severe hereditary factor X deficient patients receiving Coagadex (human factor X concentrate) for major surgical procedures.
To complete the ongoing 10-year study extension of Protocol V503-002 to evaluate the long-term safety, immunogenicity and effectiveness of Gardasil 9 in males and females who were between 9 and 15 years of age at enrollment.
Gardasil 9, Human Papillomavirus 9-valent Vaccine, Recombinant
To conduct a 10-year study extension of Protocol V503-001 to evaluate the long-term safety, immunogenicity and effectiveness of Gardasil 9 in women who were 16 to 26 years of age at enrollment.
To conduct an observational study to further characterize the safety profile of Gardasil 9 in approximately 10,000 persons.
To establish a pregnancy registry as described in your final protocol submitted September 16, 2014, to continue for at least 5 years, to prospectively collect data on spontaneously reported exposures to Gardasil 9 occurring within 30 days prior
To complete the ongoing study V503-010-01 to evaluate the persistence of antibody titers 36 months after vaccination to the nine vaccine-HPV types in GARDASIL 9 in males and females who received 2-dose and 3-dose regimens of GARDASIL 9.
To conduct the confirmatory study (V118_18) to assess efficacy of Fluad aQIV as compared to an active control Tdap vaccine (tetanus toxoid, reduced diphtheria toxoid and acellular pertussis vaccine, adsorbed [Boostrix]) in adults 65 years of ag
Deferred Pediatric Trial (V118_05) under PREA to evaluate efficacy, safety and immunogenicity of Fluad aQIV when administered to children 6 months to less than 72 months of age.
Deferred pediatric trial (V70_29) under PREA to evaluate the safety and immunogenicity of Fluad when administered to children 6 months to less than 72 months of age.
Deferred pediatric trial (V118_19) under PREA to evaluate the safety and immunogenicity of Fluad aQIV when administered to children 6 years to less than 9 years of age.
Deferred pediatric trial (V118_14) under PREA to evaluate the safety and immunogenicity of Fluad aQIV when administered to infants less than 6 months of age.
To collect additional efficacy and safety data for OBIZUR in adults with acquired hemophilia A in the Treatment Registry study under Protocol 241302, “A Non-Interventional Study of Safety and Effectiveness of Recombinant Porcine Sequence FVII
OBIZUR, Antihemophilic Factor (Recombinant), Porcine Sequence
Conduct a prospective observational cohort study of 920 IMLYGIC-treated patients to characterize the risk of herpetic infection among patients, close contacts, and healthcare providers; each subject will be followed for 5 years after initiating
Complete the ongoing single-arm trial to evaluate the biodistribution and shedding of IMLYGIC in 60 IMLYGIC-treated subjects (study Protocol #20120324).
Deferred pediatric study under PREA for use as an adjunct to surgical hemostasis for mild to moderate bleeding from small vessels when control of bleeding by standard surgical techniques is ineffective or impractical in pediatric patients ages
Deferral Extension Requested 5/31/2016. Deferral Extension Granted per FDA letter dated 09/08/2016. Study is ongoing.
To conduct the ongoing study V102_16 to assess the performance of immunologic assays for evaluating the breadth of coverage against diverse Neisseria meningitidis serogroup B strains.
To conduct study V72_72 of BEXSERO among persons 10 years through 25 years of age in the US to confirm effectiveness against a panel of diverse Neisseria meningitidis serogroup B strains.
A deferred pediatric study (V72_57) under PREA to evaluate the safety and immunogenicity of BEXSERO in North American infants 6 weeks through 12 months of age for the prevention of invasive group B meningococcal disease.
A deferred pediatric study (V72_28) under PREA to evaluate the safety and immunogenicity of BEXSERO in infants 2.5 months through 11 months of age and in children 2 years through 10 years of age for the prevention of invasive group B meningococ
The final report (STN 125546/53) was submitted to FDA on February 12, 2016.
To conduct a safety and immunogenicity study (V72_79) to assess concomitant use of BEXSERO with a second dose of Meningococcal (Groups A, C, Y and W-135) Oligosaccharide Diphtheria CRM197 Conjugate Vaccine in persons 16 years through 18 years o
To establish a pregnancy registry for BEXSERO in the US to prospectively collect data on spontaneously reported exposures to BEXSERO occurring within 30 days prior to the last menstrual period or at any time during pregnancy. You will submit an
To conduct the ongoing study (B1971009) among persons 10 years to less than 19 years of age to confirm effectiveness against a panel of diverse meningococcal group B strains.
To conduct the ongoing study (B1971016) among persons 18 years to less than 26 years of age to confirm the effectiveness against a panel of diverse meningococcal group B strains.
A deferred pediatric study (B1971017) under PREA to evaluate the safety and immunogenicity of Trumenba in children 2 years to less than 10 years of age for the prevention of invasive group B meningococcal disease.
A deferred pediatric study (B1971035) under PREA to evaluate the safety and immunogenicity of Trumenba in children 12 months to less than 24 months of age for the prevention of invasive group B meningococcal disease.
A deferred pediatric study (B1971051) under PREA to evaluate the safety and immunogenicity of Trumenba in children 1 year to less than 10 years of age for the prevention of invasive group B meningococcal disease.
To conduct the ongoing safety and immunogenicity study (B1971015) to assess concomitant use of Trumenba with Meningococcal (Groups A, C, Y and W-135) Polysaccharide Diphtheria Toxoid Conjugate Vaccine and Tetanus Toxoid, Reduced Diphtheria Toxo
To conduct the ongoing controlled study (B1971014) to further describe the safety of Trumenba in persons 10 years to less than 26 years of age.
To conduct a cohort study (B1971052) to examine pregnancy and birth outcomes following vaccination with Trumenba prior to or during pregnancy.
To conduct a study to assess safety, tolerability, and immunogenicity of Trumenba administered on a 0-, 6-month schedule in healthy subjects aged =10 to less than 26 years
A deferred pediatric study under PREA to evaluate the safety and immunogenicity of a 2-dose regimen (0 and 6 months) of Trumenba in children 1 year to less than 10 years of age for the prevention of invasive group B meningococcal disease
The study has not been initiated but does not meet the criterion for delayed.
GENA-05: Evaluation of immunogenicity, efficacy and safety of Antihemophilic Factor (Recombinant) in previously untreated patients.
Nuwiq, Antihemophilic Factor (Recombinant), rAHF
GENA-13: Evaluation of long-term immunogenicity, tolerability, and efficacy of Antihemophilic Factor (Recombinant) in previously treated children.
GENA-15: Extension for subjects who completed GENA-05.
GENA-99: Post-licensure trial to document long-term immunogenicity, safety, and efficacy of Antihemophilic Factor (Recombinant) in patients treated in normal clinical practice.
Conduct a field study (protocol AX-003A) to evaluate the efficacy, pharmacokinetics, and safety of Anthrax Immune Globulin (Human) for the treatment of toxemia associated with inhalational anthrax in adult and pediatric patients in combination
Anthrasil, Anthrax Immune Globulin Intravenous (Human)
To submit annual progress reports as well as interim clinical summary reports including available cumulative clinical and pharmacokinetic data every three years from use of Anthrax Immune Globulin (Human) in sporadic systemic anthrax cases (pro
Deferred pediatric study under PREA for the on-demand treatment and control ofbleeding episodes and routine prophylaxis to reduce the frequency of bleeding episodesin pediatric patients ages 0 to less than 12 years (A phase 3 prospective, uncon
Per FDA letter dated 22-Dec-2016, this PMR/PMC is fulfilled.
ADYNOVATE, Antihemophilic Factor (Recombinant), PEGylated
Deferred pediatric study under PREA for the treatment of perioperative management ofbleeding in pediatric patients ages two years to less than 17 years (A phase 3,prospective, open label, multi-center study of efficacy and safety of ADYNOVATE i
Deferred pediatric study under PREA for routine prophylaxis to compare the efficacy and safety of two different pharmacokinetics (PK) guided dosing regimens in pediatric patients ages 12 to less than 17 years (A phase 3, prospective, randomized
Conduct “A phase 3, prospective, open label, multi-center study of efficacy and safety of ADYNOVATE in the perioperative management of bleeding in PTPs age 2-75 years” [clinical study 261204] – ADULT COMPONENT ONLY.
Conduct “A phase 3b, prospective, open label, and multicentercontinuation study of safety and efficacy of ADYNOVATE in the routine prophylaxis of bleeding to reduce the frequency of bleeding episodes in PTPs” age 12 years and above [clinica
Conduct “A phase 3, prospective, randomized, multi-center clinical study comparing the safety and efficacy of BAX 855 [ADYNOVATE] following PK-guided prophylaxis targeting two different FVIII trough levels in subjects with severe Hemophilia A
Conduct “A phase 3, multi-center, open label study to investigate safety and immunogenicity of ADYNOVATE in previously untreated patients(PUPs)” [clinical study 261203]. This study will evaluate on-demand treatment andcontrol of bleeding ep
Collect additional safety and efficacy information of KOVALTRY in patients with hemophilia A in a clinical study in 25 previously untreated patients under Protocol 13400 “A multi-center Phase III uncontrolled open label trial to evaluate safe
KOVALTRY, Antihemophilic Factor (Recombinant), Full Length
Collect additional safety and efficacy information of KOVALTRY in patients with hemophilia A in an extension clinical study under Protocol 13400 “A multicenter Phase III uncontrolled open-label trial to evaluate safety and efficacy of BAY 81-
Deferred pediatric study (PXVX-VC-200-006) under PREA for active immunization against disease caused by V. cholerae serogroup O1 in pediatric patients ages 2 years to less than 18 years traveling to cholera-affected areas. This study will evalu
To establish a pregnancy registry for VAXCHORA in the United States to prospectively collect data on spontaneously reported exposures to VAXCHORA occurring within 28 days prior to the last menstrual period or at any time during pregnancy. You w