This quarterly review on new drug applications contains data for applications approved for the first time during the first quarter of 2020 which includes New Molecular Entities (NMEs) and new biologics. A total of 30 applications were approved by the FDA during these three months.
Following are the percentages of various dosage forms in this quarter - 30% tablets, 40% injectables, 3.3% capsules and 26.7% miscellaneous. Miscellaneous dosage forms include - ophthalmic implants, ophthalmic drops, nasal solutions, oral suspensions/solutions, oral granules and transdermal system formulations.
The FDA approved avapritinib (Ayvakit, Blueprint Medicines) to treat adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. Avapritinib is a potent and selective inhibitor of activated KIT and PDGFRA mutant kinases. In certain diseases, mutations in KIT and PDGFRA force protein kinases into an increasingly active state. Avapritinib is uniquely designed to bind and inhibit the active conformation of these proteins.
TEPEZZA (Teprotumumab-trbw, MW ~148 kilodaltons) is an insulin-like growth factor-1 receptor inhibitor indicated for the treatment of Thyroid Eye Disease. It is a fully human IgG1 monoclonal antibody produced in Chinese hamster ovary (CHO-DG44) cells. The recommended dose of TEPEZZA is an intravenous infusion of 10 mg/kg for the initial dose followed by an intravenous infusion of 20 mg/kg every three weeks for seven additional infusions.
NEXLETOL (bempedoic acid) is an adenosine triphosphate-citrate lyase (ACL) inhibitor indicated as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia or established atherosclerotic cardiovascular disease who require additional lowering of LDL-C.
The FDA approved Lactitol (Pizensy), an oral solution for treating chronic idiopathic constipation (CIC) in adults. CIC is a complex gastrointestinal disorder. It is characterized by symptoms, for which there is no identifiable cause. Lactitol is a simple monosaccharide sugar alcohol taken once per day that generates an osmotic effect in order to trigger the influx of water into the small intestine and consequently helps making CIC symptoms less intense.