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PTC Therapeutics Submits Vantiquinone NDA to FDA

Evidence of efficacy and safety data support the potential for vatiquinone to fill the significant unmet need for patients living with Friedreich ataxia.

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By: Charlie Sternberg

Associate Editor

PTC Therapeutics Inc. has submitted the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration (FDA)—marking the fourth approval application PTC has submitted to the FDA this year.   “We are excited to have reached this important milestone in the development of vatiquinone,” said Matthew B. Klein, M.D., CEO of PTC Therapeutics. “The evidence of short- and long-term efficacy as...

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