Marathon Biopharmaceuticals

Marathon Pharmaceuticals was a biopharmaceutical company based in Northbrook, Illinois, that specialized in developing and commercializing therapies for rare diseases. The company gained significant industry and political attention following the 2017 FDA approval of its lead product, Emflaza (deflazacort), for the treatment of Duchenne muscular dystrophy (DMD). Although deflazacort had been used off-label internationally for years at a low cost, Marathon announced a U.S. list price of $89,000 per year, sparking a major controversy regarding orphan drug pricing. Under pressure from patient advocacy groups and U.S. legislators, the company paused its commercial launch and eventually sold the rights to Emflaza to PTC Therapeutics for $140 million in cash and stock. Following the divestiture of its primary asset, Marathon Pharmaceuticals effectively wound down its operations, with its leadership team transitioning to focus on Paragon Biosciences.