PTC Therapeutics Submits Vatiquinone NDA To FDA For Friedreich Ataxia Treatment In Children And Adults

PTC Therapeutics, Inc. announced today that it has submitted the New Drug Application (NDA) for vatiquinone to the U.S. Food and Drug Administration (FDA). The drug is intended to treat children and adults living with Friedreich ataxia (FA). The vatiquinone NDA (New Drug Application) is supported by data from the placebo-controlled MOVE-FA study and two long-term studies involving both pediatric and adult patients with Friedreich's Ataxia (FA). The results from these studies show that vatiquinone significantly slows disease progression on important disease markers, with lasting and meaningful effects. Additionally, the studies confirm that vatiquinone is safe and well-tolerated across all age groups tested.

Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, expressed his views saying, “We are excited to have reached this important milestone in the development of vatiquinone. 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.”

The vatiquinone NDA is the fourth application PTC has submitted to the FDA this year. Earlier in 2024, PTC submitted a BLA for its AADC gene therapy in March, which was approved in November. Additionally, NDAs for sepiapterin, a treatment for adults and children with phenylketonuria (PKU), and for Translarna™ (ataluren), a treatment for boys and young men with nonsense mutation Duchenne muscular dystrophy, have also been submitted and are under review.