Posted by Michael Wonder on 11 Aug 2016
FDA accepts Marathon Pharmaceuticals’ new drug applications for deflazacort for the treatment of Duchenne muscular dystrophy and grants priority review
10 August 2016 - Deflazacort could be among the first FDA approved treatments for this devastating genetic disorder.
Marathon Pharmaceuticals today announced the New Drug Applications (NDAs) for the investigational drug deflazacort have been accepted for filing and granted Priority Review by the U.S. FDA. The NDAs (one for immediate-release tablet formulations and one for an oral suspension formulation) request approval for deflazacort for the treatment of patients with Duchenne muscular dystrophy, the most common and most severe form of muscular dystrophy.
The FDA’s priority review status is reserved for drugs that offer significant improvements over existing options or provide a treatment for a condition for which no approved treatment currently exists, and shortens the agency’s review time from 10 months to a goal of six months. A decision on the application is anticipated in February 2017 based on the Prescription Drug User Fee Act. Deflazacort previously received fast track status, orphan drug designation and rare pediatric disease status from the FDA.
