PTC Therapeutics submits NDA for vatiquinone in Friedreich ataxia

PTC Therapeutics (PTCT) submitted the vatiquinone New Drug Application, or NDA, for the treatment of children and adults living with Friedreich ataxia, or FA, to the FDA. The vatiquinone NDA is based on data from the placebo-controlled MOVE-FA study as well as results from two long-term studies including pediatric and adult FA patients. Data from these three studies demonstrate significant, durable and clinically meaningful evidence of slowing disease progression on key aspects of disease. In addition, these studies demonstrate that vatiquinone is safe and well tolerated in all age groups studied. The vatiquinone NDA is the fourth approval application PTC has submitted to the FDA this year.