DMD Drug Emflaza Acquired by PTC Therapeutics

TranslarnaToday, MDA learned that PTC Therapeutics reached an agreement with Marathon Pharmaceuticals to acquire all rights to Emflaza™ (deflazacort), the first treatment approved in the U.S. for all Duchenne muscular dystrophy (DMD) patients five years and older, regardless of their genetic mutation. Emflaza was approved for use earlier this year by the FDA though the commercial roll-out of the drug was halted pending concerns about its costs. MDA will continue to advocate on behalf of its families to ensure they receive the treatment they need, and we look forward to working with PTC and the wider Duchenne community on this issue.

Marathon also issued a letter to the DMD community about this news that can be read here.