PTC Therapeutics Announces Vatiquinone NDA Submission to FDA for the Treatment of Children and Adults Living with Friedreich Ataxia 12/19/2024 08:00AM ET - If approved, vatiquinone would be the first and only authorized therapy for children with FA - - PTC's fourth approval application submitted to FDA in 2024 - WARREN, N.J., Dec. 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today the submission of 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). "We are excited to have reached this important milestone in the development of vatiquinone," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The evidence of short- and long-term efficacy as well as the extensive safety data collected, particularly in children, supports the potential for vatiquinone to fill the significant unmet need for children living with Friedreich ataxia as well as provide a potential treatment option for adults living with FA."