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).
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.
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