PTC Therapeutics (NASDAQ: PTCT) announced on February 12, 2026 that it has withdrawn the New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy (DMD) following feedback from the U.S. Food and Drug Administration.
According to the company, FDA reviewers indicated the NDA data are unlikely to meet the Agency's threshold of substantial evidence of effectiveness to support approval, and PTC therefore withdrew the submission; leadership expressed disappointment after more than two decades of development.
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