PTC Therapeutics Files NDA With US FDA for Vatiquinone to Treat Friedreich's Ataxia

MT Newswires Live12-19

PTC Therapeutics (PTCT) said Thursday it has filed a new drug application with the US Food and Drug Administration for vatiquinone to treat children and adults with Friedreich's ataxia, a rare neuromuscular disorder.

The application is supported by data from the placebo-controlled MOVE-FA study and two long-term studies that show a "significant" reduction in disease progression and demonstrate a "safe and well-tolerated" vatiquinone across all age groups, the company said.

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