The Committee will discuss a supplemental new drug application for VICTOZA (liraglutide) injection (sNDA 022341), sponsored by Novo Nordisk, for the proposed additional indication of: as an adjunct to standard treatment of cardiovascular risk factors to reduce the risk of major adverse cardiovascular events (cardiovascular death, non-fatal myocardial infarction, or non-fatal stroke) in adults with type 2 diabetes mellitus and high cardiovascular risk.
The Commitee will discuss potential pediatric development plans/written requests for: (1) APX - 005M, presentation by Apexigen, Inc.; (2) PMO1183 ( lurbinectedin ), presentation by PharmaMar USA Inc.; (3) ASP2215 ( gilteritinib ), presentation by Astellas Pharma Global Development, Inc. (4) Prexasertib , presentation by Dista Products/Eli Lilly and Company and (5) olaratumab , presentation by Eli Lilly and Company.
The Committee will discuss and make recommendations on the safety and efficacy of a Hepatitis B Vaccine manufactured by Dynavax.
The Committee 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pertaining to the new “Common Rule” (the Federal Policy for the Protection of Human Subjects) and recent FDA Draft Guidance regarding the use of real-world evidence to support regulatory decision-making for medical devices.
The Committee supported, by a nearly unanimous vote of 14-Yes to 1-No, with no abstentions, the licensure of “Epoetin Hospira” as a biosimilar product to US-licensed Epogen/Procrit.
The Committee supported by a vote of 12-Yes to 4-No, with no abstentions, the risk-benefit profile of neratinib maleate by Puma Biotechnology (Puma) for the extended adjuvant treatment of human epidermal growth factor receptor 2 (HER2)-overexpressed/amplified in adult breast cancer patients who have received prior therapy with trastuzumab (US trade name Herceptin).
In the afternoon session, the Committee supported, by a vote of 10-Yes to 3-No, with no abstentions, the risk-benefit profile of L-glutamine by Emmaus Medical, Inc. (Emmaus) for the treatment of Sickle Cell Disease.
The Committees jointly supported, by a vote of 14-Yes to 0-No, with no abstentions, the use of an indwelling central venous access devices, in certain circumstances, in the clinical trial “A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients with Duchenne Muscular Dystrophy (ESSENCE).”
The Committee discussed considerations for evaluation of Respiratory Syncytial Virus vaccine candidates in seronegative infants.
The Committee met to consider Spinal Muscular Atrophy (SMA) deficiency for a full evidence review for addition to the Recommended Uniform Screening Panel (RUSP). In addition, the Committee heard updates on topics related to newborn screening (NBS) and medical foods.
The Committee supported, by a vote of 11-Yes to 0-No, with no abstentions, the safety and efficacy of 5-aminolevulinic acid (5-ALA), powder for oral solution, by NX Development Corp. (NXDC) for use as an imaging agent during glioma surgery.
The Board met to provide recommendations on the Agency’s work plan on the allocation of innovation funds. Section 1002 of the 21st Century Cures Act provides $495 million in funding to the FDA from fiscal year (FY) 2017 through FY 2025 for FDA Innovation Projects. The Act requires the FDA Commissioner to submit a work plan that describes the budget for planned activities and provides justification based on advancing public health. The plan must be submitted by June 11, 2017, which is within 180 days of the bill’s passage (December 13, 2016). The Act states that prior to submitting the work plan to Congress, the Commissioner must seek recommendations from the SBFDA and include these recommendations in the work plan.
The Committee discussed and voted on substances that were nominated for the list of bulk drug substances allowed for compounding and on products that were nominated for the difficult to compound list (as per sections 503A and 503B of the Food, Drug & Cosmetic Act).
· A majority of the Committee members agreed with the FDA’s recommendation to exclude the following 6 bulk drug substances on the substances allowed list: nicotinamide adenine dinucleotide, nicotinamide adenine dinucleotide disodium reduced, nettle, ubiquinol, vanadyl sulfate, and artemisinin.
· A majority of the Committee members agreed with the FDA’s recommendation to include oral solid modified release drug products that employ coated systems on the products that are difficult to compound list.
· The Committee also heard an update from the FDA in regard to inspections.
The Committee discussed the development of antibacterial drugs that treat a single species of bacteria when the target species infrequently causes infections. Overall, the Committee supported the conduct of non-inferiority trials with expanded margins to support approval. The Committee did not support the use of data obtained from studies of animal models as a sole basis for approval.
Drug Safety and Risk Management Advisory Committee and the Anesthetic and Analgesic Drug Products Advisory Committee
The Committees jointly supported, by a vote of 19-Yes to 0-No, with one abstention, the approval of RoxyBond, oxycodone immediate-release (IR) tablets, submitted by Inspirion Delivery Sciences, LLC (IDS) with the proposed indication of management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate.
A majority of the Committees, 19 of 20 members, voted that, if approved, RoxyBond should be labeled as an abuse-deterrent product by the IN route of abuse.
A majority of the Committees, 16 of 20 members, voted that, if approved, RoxyBond should be labeled as an abuse-deterrent product by the IV route of abuse.
The Committee met to discuss Novo Nordisk’s Biologics License Application (BLA 125611), for Recombinant Human Coagulation Factor IX, GlycoPEGylated. The product is also referred to as nonacog beta pegol or N9-GP.
In a separate Tuesday afternoon session, the Committee heard a summary of responses to Docket FDA-2016-N-1502: Blood Donor Deferral Policy for Reducing the Risk of Human Immunodeficiency Virus Transmission by Blood and Blood Products.
On Wednesday, April 5, 2017, the Committee heard presentations on the research programs in the Laboratory of Emerging Pathogens in the Division of Emerging Transfusion-Transmitted Diseases, Office of Blood Research and Review, Center for Biologics Evaluation and Research, FDA.
The Committees did not support, by a vote of 5-Yes to 15-No, with no abstentions, that the combination of an analgesic with an antacid is a rational combination for the over-the-counter (OTC) use to relieve minor aches and pains associated with heartburn, sour stomach, acid indigestion, fullness, belching, gas, or nausea. The Committees also expressed concerns with the hangover indication of several OTC monographs.
The Committee unanimously supported, by a vote of 11-Yes to 0-No, with no abstentions, the safety and efficacy of rituximab/hyaluronidase injection for subcutaneous use, by Genentech, Inc. (Genentech), to treat patients with certain types of lymphoma and leukemia.
The goal date for the FDA to decide on approval (the PDUFA date) is June 26, 2017.
The Committee will review recommendations pertaining to the Common Rule and the use of real-world evidence for devices.
The use of model-informed drug development (MIDD) for new and generic drugs has significantly increased over the past several years. The Committee discussed strategies, approaches, and challenges in MIDD with specific focus on two areas. During the morning session, the Committee discussed approaches and evidentiary information needed for applying physiologically-based pharmacokinetic modeling and simulation throughout a drug’s lifecycle. During the afternoon session, the Committee discussed mechanistic model informed safety evaluation with a focus on drug potential for causing arrhythmias. The Comprehensive in Vitro Proarrhythmia Assay was discussed as an exemplar.
Drug Safety and Risk Management Advisory Committee (DSRM) and the Anesthetic and Analgesic Drug Products Advisory Committee (AADPAC)
The Committees discussed safety issues for new drug application (NDA) 201655, OPANA ER (oxymorphone hydrochloride) Extended-release Tablets, by Endo Pharmaceuticals Inc., with the indication of management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. The product is an approved extended-release (ER) formulation intended to have abuse-deterrent properties based on its physicochemical properties, however, this information is not currently reflected in product labeling.
A majority of the Committee, 18 of 27 members, voted that the benefits of reformulated OPANA ER do not continue to outweigh its risks.
The Committee will meet to discuss and make recommendations on the selection of strains to be included in the influenza virus vaccines for the 2017-2018 influenza season.
The Committee will discuss pediatric-focused safety reviews, as mandated by the Best Pharmaceuticals for Children Act, the Pediatric Research Equity Act and the Pediatric Medical Device Safety and Improvement Act .
On March 6, 2017, the Committee will discuss the following products (listed by FDA Center):
Center for Drug Evaluation and Research (CDER)
a. NITROPRESS (sodium nitroprusside)
b. KUVAN (sapropterin dihydrochloride)
FDA will provide the analysis of a possible safety signal regarding the use of the drug product EXJADE (deferasirox) in children with fever and dehydration, which was discussed at the September 2015 meeting.
The Committee will also discuss the role of pharmacogenomics in pediatric product development.
On March 7, 2017, the Committtee will discuss the following products (listed by FDA Center):
Center for Biologics Evaluation and Research (CBER)
a. EPICEL (cultured epidermal autografts) (humanitarian device exemption (HDE))
b. NOVOEIGHT (Antihemophilic Factor (Recombinant))
c. RIXUBIS Coagulation Factor IX (Recombinant)
Center for Devices and Radiological Health (CDRH)
a. IMPELLA RP SYSTEM (HDE)
b. LIPOSORBER LA-15 SYSTEM (HDE)
c. MEDTRONIC ACTIVA DYSTONIA THERAPY (HDE)
The Committee will discuss new drug application (NDA) 209241, Valbenazine 40 mg capsules, for the proposed treatment of Tardive Dyskinesia, submitted by Neurocrine Biosciences, Inc.
The ACHDNC heard presentations and held discussions on topics related to newborn screening (NBS) 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 also heard updates from the following workgroups:
· Education and Training Workgroup;
· Laboratory Standards and Procedures Workgroup; and
· Follow-up and Treatment Workgroup.
The ACHDNC did not vote on a proposed addition of a condition to the Recommended Uniform Screening Panel (RUSP)
The Committee dicussed appropriate clinical trial design features, including acceptable endpoints for demonstrating clinical benefit, for drugs intended to treat secondary hypogonadism while preserving or improving testicular function, including spermatogenesis
The Committee reviewed topics related to the safety of donated blood.
The Board heard reports from the FDA and discussed topics including: 1. Strategic goals for regulatory science at the Center for Biologics Evaluation and Research (CBER); 2. Progress updates on FDA’s Opioid Action Plan and the Bovine Heparin Initiative; 3. Response from the Office of Scientific Professional Development to the Science Board’s report on the Commissioner’s Fellowship Program; 4. Report from the Scientific Engagement Subcommittee; and 5. Report from the Food Emergency Response Network Cooperative Agreement Program Evaluation Subcommittee.
The Committee discussed and made recommendations on FDA’s draft Strategic Plan for Risk Communication and Health Literacy.
The Committee narrowly supported, by a vote of 7-Yes to 6-No, with no abstentions, the safety and efficacy of solithromycin capsules and solithromycin for injection, sponsored by Cempra Pharmaceuticals, Inc. (Cempra), for the proposed indication of the treatment of community-acquired bacterial pneumonia (CABP).
The Committee discussed five bulk drug substances nominated for inclusion on the section 503A Bulks List.
- Diindolylmethane - Treatment of cancer
- Glycolic acid - Hyperpigmentation (including melasma) and photodamaged skin
- Trichloroacetic acid - Common warts and genital warts
- Kojic acid - Hyperpigmentation and as a chelating agent to promote wound healing
- Vasoactive intestinal peptide - A condition described as “chronic inflammatory response syndrome”
The Committee also discussed drug products that employ transdermal and topical delivery systems, which were nominated for the Difficult to Compound List.
The Committee met to consider Guanidinoacetate Methyltransferase (GAMT) deficiency for a full evidence review for addition to the Recommended Uniform Screening Panel (RUSP). In addition, the Committee heard updates on topics related to newborn screening and medical foods.
The Committee will 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).
The Committee heard updates of research programs in the Laboratory of Immunobiochemistry of the Division of Bacterial, Parasitic and Allergenic Products (DBPAP), Office of Vaccines Research and Review (OVRR), Center for Biologics Evaluation and Research (CBER), FDA. The open session portion of the meeting occurred by teleconference from 1:00 PM – 2:20 U.S. EDT.