PTC Therapeutics announced on October 30, 2024, that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the New Drug Application (NDA) for Translarna (ataluren). This application is for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
The NDA resubmission is based on findings of significant benefit demonstrated in the ITT population (N=359) of the global placebo-controlled trial Study 041. Following 72 weeks of Translarna treatment, significant benefits were recorded on key study endpoints such as the six-minute walk distance (p=0.0248) and NorthStar Ambulatory Assessment (p=0.0283).
Additionally, the NDA includes findings from the STRIDE registry, which showed a 3.5-year delay in loss of ambulation (p<0.0001) and a 1.8-year delay in reaching a predicted forced vital capacity of less than 60% (p=0.0028). As this is an NDA resubmission following a complete response letter, the FDA is not obligated to follow PDUFA review timelines, and an action date has not been provided.
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