Marathon’s DMD Drug Granted Rare Pediatric Disease Designation

The FDA has granted rare pediatric disease designation to another investigational drug to treat Duchenne muscular dystrophy, this time to Marathon Pharmaceuticals’ deflazacort.

The Northbrook, Ill., drugmaker expects to submit an NDA for the product in the first half of 2016. Deflazacort previously received orphan drug designation and fast-track status.

Phase 3 clinical studies have been completed, and a final bioavailability study in healthy volunteers treated with the final formulation will be completed by the end of this year, Timothy Cunniff, Marathon’s executive vice president of research and development, said.

Other potential DMD treatments under development include Catabasis Pharmaceuticals’ CAT-1004, Sarepta Therapeutics’ eteplirsen and BioMarin Pharmaceuticals’ drisapersen, which all earned rare pediatric disease designations in recent months, and Tarix Orphan’s TXA127, which won fast-track designation earlier this week.

Companies receiving approval of an NDA or BLA for a rare pediatric disease therapy may be eligible for a voucher that can be used to obtain priority review of a different product. — Jonathon Shacat