PTC Therapeutics receives Refusal to File letter from FDA for Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy,

PTC Therapeutics, Inc. announced that it received yesterday evening a Refuse to File letter from the FDA regarding PTC's New Drug Application (NDA) for Translarna (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The FDA states in the Refuse to File letter that the application was not sufficiently complete to permit a substantive review. PTC first learned of the Refuse to File decision via this letter and is reviewing its content to determine the appropriate next steps.

Comment: Ataluren in its Phase III study failed to prove that it could help the average patient walk significantly further than a placebo.