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news.BioCryst Pharmaceuticals, announced that the U.S. Food and Drug Administration has granted Fast Track designation for BCX4161, a novel, orally administered, selective inhibitor of plasma kallikrein in advanced clinical development for the treatment of hereditary angioedema.
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The Fast Track designation process of the FDA is designed to facilitate the development and expedite the review and approval of drugs intended to treat serious or life threatening conditions and that address unmet medical needs. A drug that receives Fast Track designation is usually eligible for more frequent written communication and meetings with the FDA to discuss the drug’s development plan and the collection of appropriate data supporting drug approval.
Priority Review and Rolling Review may be granted, if relevant criteria are met. Rolling Review allows a drug company to submit completed sections of its New Drug Application (NDA) for review by FDA on an ongoing basis, rather than wait until the entire NDA is completed and then reviewed.
"We are very pleased to have been granted orphan drug and fast track status from the FDA, as well as recently receiving a positive opinion for orphan drug designation in Europe," said Jon P. Stonehouse, President & Chief Executive Officer of BioCryst.
"BCX4161 and our second generation molecules have the potential to significantly improve HAE patient treatment and their quality of life. We look forward to reporting results from OPuS-2 and sharing updates regarding BCX7353 and our other second generation HAE assets during 2015."
BioCryst is enrolling HAE patients in the OPuS-2 trial of BCX4161; a double-blind, randomized, placebo controlled trial conducted in the U.S. and certain EU countries, with the goal of demonstrating the efficacy and safety of BCX4161 treatment for 12 weeks in approximately 100 patients with HAE. BioCryst expects to report results from OPuS-2 by the end of 2015.
Discovered by BioCryst, BCX4161 is a novel, selective inhibitor of plasma kallikrein in development for prevention of attacks in patients with HAE. By inhibiting plasma kallikrein, BCX4161 suppresses bradykinin production.
Bradykinin is the mediator of acute swelling attacks in HAE patients. BCX4161 was granted orphan drug status by the FDA and was issued a positive opinion on the application for orphan drug designation for the treatment of patients with HAE by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). BioCryst is also advancing BCX7353 and other second oral kallikrein inhibitors for HAE through preclinical development.