PTC Receives Refuse to File Letter from FDA for Translarna™ (ataluren)

PTC Therapeutics, Inc. announced that it received yesterday evening a Refuse to File letter from the United States Food and Drug Administration (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).

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The FDA states in the Refuse to File letter that the application was not sufficiently complete to permit a substantive review.

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PTC first learned of the Refuse to File decision via this letter and is reviewing its content to determine the appropriate next steps.