Marathon Readies DMD Drug for FDA

While much of the attention of the Duchenne Muscular Dystrophy community has been focused on the recent FDA advisory committee review of Sarepta Therapeutics’ experimental drug Eteplirsen, Marathon Pharmaceuticals has been working to advance its own DMD drug candidate to the FDA. The company expects this month to file an application with the agency for approval to market Deflazacort, a corticoid steroid. We spoke to Tim Cunniff, head of research and development for Marathon Pharmaceuticals, about why the company thinks Deflazacort represents an improvement over existing therapies, what the company has learned from Sarepta’s experience, and its new effort to develop a precision medicine program in Duchenne.