PTC announces FDA acceptance, priority review of NDA for vatiquinone

PTC Therapeutics (PTCT) announced that the U.S. Food and Drug Administration, FDA, has accepted for filing the New Drug Application, NDA, for vatiquinone for the treatment of children and adults living with Friedreich’s ataxia. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act, PDUFA, target action date of Aug. 19, 2025. “We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich’s ataxia,” said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. “If approved, vatiquinone would be the first therapy for pediatric patients with FA, and provide a potential safe, well-tolerated and effective treatment alternative for adults. The granting of priority review by FDA reflects the significant unmet need for younger patients with FA. We look forward to working collaboratively with FDA during the review process.”