PTC Therapeutics withdraws Translarna NDA

13 February 2026

USA-based rare disease drug developer PTC Therapeutics (Nasdaq: PTCT) saw its shares drop 5.2% to $67.05 after market close, when it announced that it has withdrawn the New Drug Application (NDA) resubmission for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following US Food and Drug Administration (FDA) feedback on the application review.

The withdrawal represents a major setback for PTC Therapeutics regarding its US commercialization efforts for Translarna.

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