RARECast: Marathon Preps 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 for the FDA. The company expects to file an application with the agency for approval this month to market Deflazacort, a corticoid steroid.

We spoke to Tim Cunniff, head of research and development for Marathon Pharmaceuticals, about it’s new effort to develop a precision medicine drug for Duchenne, why the company thinks Deflazacort represents an improvement over existing therapies and what the company has learned from Sarepta’s experience.