This column summarizes New Drug Applications (NDAs) approved in the fourth quarter 2015 (November-December 2015). In these two months, FDA approved 30 NDAs.
Fifteen (50%) of NDAs were for small molecules while the remaining were large molecules. As the trend has been observed in recent times, pharmaceutical companies are working more and more on large molecules. In this quarter, 16 companies (53.3%) receiving NDA approvals were large companies and the remaining 14 companies (46.6 %) were mid-sized and small companies. The number of large and small companies which obtained NDAs approved for large molecules were eight and seven, respectively. Companies such as AstraZeneca, Bristol Myers Squibb, Eli Lilly, Eagle Pharmaceuticals, Baxalta Inc., Gilead Sciences, Genentech Inc., Boehringer Ingelheim, Merck Inc. , received 2 NDAs each in this quarter. Injections were the preferred dosage form (60%). Most of the large molecules were delivered as injections. Only two out of 30 (6.6%) of NDAs were combination dosage forms, out of which Genvoya (indicated for HIV infection) had 4 different drugs. Ten drug molecules were granted Orphan Drug status (33.3 %).
A total of 9 out of 30 (30%) of NDA’s were granted a priority review status while 6 (20%) breakthrough status. Two out of 30 (6.6%) obtained accelerated approval and three out of 30 (10%) obtained fast track approval.
The U.S. Food and Drug Administration (FDA) approved Harvoni (Ledipasvir/Sofosbuvir) for expanded use in patients with genotypes 4, 5 and 6 chronic hepatitis C virus (HCV) infection and in patients co-infected with HIV.
BioThrax was the first vaccine to receive approval based on the Animal Rule. The Animal Rule allows animal efficacy data to be used as a basis for approval when human efficacy studies are not ethical or feasible.
Genvoya carries a Boxed Warning alerting patients and health care providers that the drug can cause a buildup of lactic acid in the blood and may pose severe liver problems, both of which can be fatal. The Boxed Warning also states that Genvoya is not approved to treat chronic hepatitis B virus infection.
Adynovate uses the Baxject II Hi-Flow needleless transfer device. Emplicilti (Elotuzumab) is the third drug for multiple myeloma approved this year and provides patients with a new oral treatment that slows disease progression when other therapy has failed. FDA approved Farydak (Panobinostat) in February and Darzalex (Daratumumab) earlier this month.
Fluad, a trivalent vaccine produced from three influenza virus strains (two subtype A and one type B), is approved for the prevention of seasonal influenza in people 65 years of age and older. Fluad, is manufactured using an egg-based process and is formulated with the adjuvant MF59, an oil-in-water emulsion of squalene oil.
Otiprio is a single-dose, physician-administered antibacterial and the first product approved by the FDA for this indication. Otiprio combines a thermosensitive gel with drug micro particles to enable single dose treatment by a physician
Vastogard is the first-of-its-kind therapy that can potentially save lives following overdose or life-threatening toxicity from chemotherapy agents.
Fast track - A process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
Breakthrough therapy - A process designed to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy.
Accelerated approval - These regulations allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint.
A Priority Review designation means FDA’s goal is to take action on an application within 6 months.