The Committee will discuss a new drug application for oral testosterone undecanoate capsules, submitted by Clarus Therapeutics, for the proposed indication of testosterone replacement in males for conditions associated with a deficiency or absence of endogenous tes osterone: Primary hypogonadism (congenital or acquired) and hypogonadotropic hypogonadism (congenital or acquired).
The Committee will discuss a new drug application for oral testosterone undecanoate capsules, submitted by Lipocine Inc. for the proposed indication of testosterone replacement in males for conditions associated with a deficiency or absence of endogenous testosterone: primary hypogonadism (congenital or acquired) and hypogonadotropichypogonadism (congenital or acquired).
The Committee will discuss a new drug application for ciprofloxacin dispersion for inhalation, sponsored by Aradigm Corp., for the proposed indication of treatment of non-cystic fibrosis bronchiectasis patients with chronic lung infections with Pseudomonas aeruginosa.
The Committee discussed appropriate patient selection criteria and clinical trial design features, including acceptable endpoints, for demonstrating clinical benefit for drugs intended to treat interstitial cystitis and bladder pain syndrome. The Committee also discussed whether bladder pain syndrome and interstitial cystitis reflect overlapping or different populations, and whether it is appropriate to assess efficacy in the same way for both conditions.
The Committee discussed and voted on the following topics:
· Bacterial risk control strategies for blood collection establishments and transfusion services to enhance the safety and availability of platelets for transfusion;
· Device classification of human leukocyte antigen, human platelet antigen, and human neutrophil antigen devices; and
· Strategies to reduce the risk of transfusion-transmitted Zika virus.
The Committee also heard information about the April 6, 2017 FDA public workshop on emerging tick-borne diseases and blood safety, as well as the Transfusion Transmissible Infections Monitoring System.
The Committee reviewed substances nominated for the list of bulk substances allowed for compounding (a.k.a. the 503A Bulks List of the FD&C Act) and products nominated for the difficult to compound list (a.k.a., the Difficult to Compound List under sections 503A and 503B of the FD&C Act).
· A majority of the Committee members agreed with the FDA’s recommendation to exclude the following four bulk drug substances from the list of bulk substances allowed for compounding: astragalus extract 10:1, 7-keto dehydroepiandrosterone, epigallocatechin gallate, and resveratrol.
· A majority of the Committee members agreed with the FDA’s recommendation to include L-citrulline on the list of bulk substances allowed for compounding.
· A slight majority of the Committee members did not agree with the FDA’s recommendation to exclude pregnenolone from the list of bulk substances allowed for compounding.
· A majority of the Committee members agreed with the FDA’s recommendations to include liposome drug products and drugs produced using hot melt extrusion on the products that are demonstrably difficult to compound list.
On Thursday, November 16, 2017 the Antimicrobial Drugs Advisory Committee (AMDAC) did not support, by a vote of 6-Yes to 9-No, with no abstentions the safety and efficacy of the proposed 14-day regimen of ciprofloxacin dry powder inhaler (DPI), sponsored by Bayer HealthCare Pharmaceuticals, Inc. (Bayer), for the proposed indication of reduction of exacerbations in non-cystic fibrosis bronchiectasis (NCFB) adult patients with respiratory bacterial pathogens. The Committee did not support, by a vote of 1-Yes to 14-No, with no abstentions the proposed 28-day regimen.
On Wednesday to Thursday, November 8-9, 2017, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) met to discuss issues related to newborn screening (NBS) and the diagnosis and treatment of individuals with heritable disorders.
The Committee discussed and made recommendations on the clinical development plan for Pfizer’s investigational Staphylococcus aureus vaccine intended for pre-surgical prophylaxis in elective orthopedic surgical populations.
Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee
The Committees supported approval of some of the proposed doses of buprenorphine subcutaneous injection (CAM2038), submitted by Braeburn Pharmaceuticals, Inc., (Braeburn), for the treatment of opioid dependence.
The Committee met to hear updates from the Centers for Disease Control and Prevention (CDC), the Centers for Medicare and Medicaid Services (CMS), and the Food and Drug Administration (FDA). In addition, the Committee heard presentations on laboratory testing in the era of telemedicine, antibiotic resistance testing issues, and culture independent diagnostic tests (CIDTs). The Committee also heard and discussed recommendations on sections of the Institute of Medicine (IOM) Report on Improving Diagnosis in Health Care, including IOM Workgroup topics of pathologists as integral care team member and interoperability.
Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee
The Committees supported, by a vote of 18-Yes to 1-No, with no abstentions, the approval of buprenorphine subcutaneous injection (RBP-6000), submitted by Indivior Pharmaceuticals, Inc. (Indivior), for the treatment of opioid dependence.
The Committee supported, by a vote of 16-Yes to 0-No, with one abstention, the safety and efficacy of semaglutide injection, submitted by Novo Nordisk, as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus.
The Committee met to discuss topics related to human research protections.
The Committee supported, by a vote of 9-Yes to 1-No, with no abstentions, the safety and efficacy of Rhopressa (netarsudil ophthalmic solution 0.02%), submitted by Aerie Pharmaceuticals Inc. (Aerie), for the proposed indication to reduce elevated intraocular pressure in patients with open-angle glaucoma (OAG) or ocular hypertension (OHT).
The Committee unanimously supported, by a vote of 16-Yes to 0-No, with no abstentions, the safety and efficacy of voretigene neparvovec by Spark Therapeutics, Inc. (Spark). The proposed indication is the treatment of patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy. The proposed trade name is Luxturna. The FDA’s goal date for a decision (PDUFA date) is January 12, 2018.
The Committee agreed unanimously with the recommendations of the World Health Organization (WHO) in regard to the strains to include in influenza virus vaccines for the 2018 southern hemisphere influenza season.
The Committee met to discuss the proposed use of Translarna (ataluren) by PTC Therapeutics (PTC) for the treatment of dystrophinopathies resulting from nonsense mutations in the dystrophin gene, including nonsense mutation Duchenne Muscular Dystrophy (nmDMD). A majority of the Committee, 10 of 11 members, voted that “although it is possible that ataluren may be effective, the data are inconclusive, and more work would be needed to establish whether ataluren is effective.”
The Committee split their vote, by a vote of 6-Yes to 6-No, with no abstentions, on the benefit-risk profile of Sutent (sunitinib malate), submitted by C.P. Pharmaceuticals International CV, represented by Pfizer, Inc. (authorized US agent) for the proposed indication of adjuvant treatment of adult patients at high risk of recurrent renal cell carcinoma (RCC) following nephrectomy.
Anesthetic and Analgesic Drugs Advisory Committee and Drug Safety and Risk Management Advisory Committee
The Committees discussed a supplemental new drug application (sNDA) for Butrans (buprenorphine transdermal system, BTDS) submitted by Purdue Pharma L.P. (Purdue), evaluating Butrans in pediatric patients aged 7 through 16 years for management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. The Committees were specially asked to discuss the findings of the clinical study of Butrans conducted in pediatric patients, and whether they support additional labeling.
The Committee (VRBPAC) supported, by unanimous votes of 11-Yes to 0-No, with no abstentions, the safety and effectiveness of Zoster Vaccine Recombinant, Adjuvanted, by GlaxoSmithKline Biologicals (GSK). The proposed trade name is Shingrix.
On Monday, September 11, 2017, the Pediatric Advisory Committee (PEDAC) discussed and voted on the use of prescription opioid products for the treatment of cough in pediatric patients.
On Tuesday, September 12, 2017, the Committee discussed and voted on pediatric-focused safety reviews for certain products that are mandated by the Best Pharmaceuticals for Children Act (BPCA), the Pediatric Research Equity Act (PREA), and the Pediatric Medical Device Safety and Improvement Act (PMDSIA).
The Committee discussed the potential risk of gadolinium retention in the brain and other body organs in patients receiving gadolinium-based contrast agents (GBCAs) for magnetic resonance imaging (MRI) procedures. A majority of the Committee agreed with the plans that were proposed by the FDA to address this potential risk, which included labeling and potentially requiring further additional safety studies.
The Committee met to hear presentations and conduct discussions on topics related to newborn screening activities, technologies, policies, guidelines, and programs for effectively reducing morbidity and mortality in newborns and children having, or at risk for, heritable disorders.
The Committee supported by a vote of 10-Yes to 1-No, with no abstentions, the approval of XELJANZ (tofacitinib) tablets and XELJANZ XR (tofacitinib) extended-release (XR) tablets, submitted by Pfizer Inc. (Pfizer), for the treatment of adult patients with active psoriatic arthritis.
The Committee voted against recommending approval, by a vote of 1-Yes to 12-No, with no abstentions, of sirukumab injection (proposed trade name PLIVENSIA), by Janssen Biotech, Inc. (Janssen), for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response or are intolerant to one or more disease-modifying anti-rheumatic drugs (DMARDs).
The Committee supported, by a vote of 12-Yes to 1-No, with 3 abstentions, the safety of Heplisav-B [Hepatitis B Vaccine, Recombinant (Adjuvanted)] by Dynavax Technologies Corporation when administered to adults aged 18 years and older. The FDA’s goal date to decide on whether to approve Heplisav-B is August 10, 2017.
Anesthetic and Analgesic Drugs Advisory Committee and Drug Safety and Risk Managment Advisory Committee
The Committees voted that Rexista (oxycodone extended-release tablets), submitted by Intellipharmaceutics Corp. (Intellipharmaceutics) should not be approved, by a vote of 1-Yes and 22-No,. The proposed indication of Rexista is the management of moderate-to-severe pain when a continuous around-the-clock analgesic is needed for an extended period of time. The Committees also voted in regard to the company’s proposed label claim regarding abuse-deterrence. The FDA’s decision goal date for the Rexista New Drug Application (NDA) is September 25, 2017.
On Tuesday and Wednesday, July 25-26, 2017, the Secretary’s Advisory Committee on Human Research Protections (SACHRP) met to discuss topics related to human research protections. The Committee reviewed recommendations pertaining to the new Common Rule, the Federal Policy for the Protection of Human Subjects under 45 CFR, Part 46.
In the morning session, the Committee supported, by a vote of 17-Yes to 0-No, with no abstentions, the licensure of ABP-215 as a biosimilar to Genentech/Roche’s Avastin (bevacizumab), submitted by Amgen Inc. (Amgen). Amgen has developed ABP-215 in collaboration with Allergan.
In the afternoon session, the Committee supported, by a vote of 16-Yes to 0-No, with no abstentions, the licensure of MYL-1401O as a biosimilar to Genentech Inc.’s Herceptin (trastuzumab), submitted by Mylan GmbH (Mylan). Mylan is developing MYL-1401O in collaboration with Biocon Ltd.
The Committee supported, by a vote of 10-Yes to 0-No, with no abstentions, the safety and efficacy of tisagenlecleucel by Novartis Pharmaceuticals Corp. (Novartis) for the treatment of pediatric and young adult patients 3 to 25 years of age with relapsed/refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL).
The PDUFA date, which is the FDA’s goal date to make an approval decision, is October 3, 2017.
The Committee supported, by a vote of 6-Yes to 1-No, with no abstentions, the safety and efficacy of Mylotarg (gemtuzumab ozogamicin) by Wyeth Pharmaceuticals Inc. (Wyeth), a subsidiary of Pfizer Inc., for the treatment of certain patients with acute myeloid leukemia (AML).