This quarterly review of new drug applications contain data for applications approved for the first time during the fourth quarter of 2019 which includes New Molecular Entities (NMEs) and new biologics. A total of 44 applications were approved by FDA during these three months. Out of 44, 16 were NMEs (36.4%) and seven were BLAs (Biologics License Applications) (15.9%).
The approvals in 4Q19 were divided into 13 different dosage forms. Following are the percentages of those dosage forms - 25% solution, 20% tablets, 18% injectable, 11% capsule, 7% powder, and the remaining 19% miscellaneous. Miscellaneous dosage forms include aerosol, cream, film, foam, gas, implant, lotion and subcutaneous systems.
Aquestive Therapeutics is a specialty pharmaceutical company that applies innovative technology to solve therapeutic problems and improve medicines for patients. On November 22, 2019, the U.S. Food and Drug Administration granted an early-action approval to the company’s Exservan oral film. The company received full FDA approval for Exservan in advance of their PDUFA action date. Exservan (riluzole), which received an Orphan Drug designation in January 2018, is an oral film formulation of the approved glutamate inhibitor - riluzole for the treatment of patients with Amyotrophic Lateral Sclerosis (ALS) patients who have difficulty swallowing. Exservan will provide help to ALS patients who have trouble swallowing as it can be administered safely and easily, twice daily, without water.
Genosyl is the first and only FDA approved tankless inhaled delivery system for nitric oxide. Persistent pulmonary hypertension of the newborn (PPHN) occurs when an infant's circulation continues to flow as it did while in the uterus once the baby is born. The result is too much blood flow bypasses the baby's lungs inhibiting their ability to breathe effectively. Inhaled nitric oxide is recommended as a first-line vasodilator therapy for PPHN, providing improved oxygenation. VERO Biotech's GENOSYL is a truly innovative way to create inhaled nitric oxide without tanks.
Sarepta Therapeutics received FDA approval for Vyondys 53 (Golodirsen), exon- skipping therapy developed to treat Duchenne muscular dystrophy (DMD). DMD is caused by a mutation or error in the DMD gene, which encodes for dystrophin, an essential protein involved in muscle cell integrity. Without dystrophin, the muscle’s integrity is lost, and it becomes easily damaged during normal activity. Vyondys 53 is designed to mask exon 53 in the mRNA (a temporary copy of a gene used to make a protein) of the DMD gene so the protein synthesis machinery can skip this exon and piece together the remaining exons to make a smaller, but functional, dystrophin protein.
Brukinsa (zanubrutibib) is BeiGene’s first drug approved in the US. It is a kinase inhibitor indicated for the treatment of adult patients with mantle cell lymphoma, who have received at least one prior therapy. Based on the overall response rate, it received an accelerated approval. BeiGene was started as a research and development company in Beijing in 2010 and began the first global Phase 3 registrational trial of zanubrutinib in 2017.