The U.S. Food and Drug Administration (FDA) has accepted for filing PTC Therapeutics’ New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich’s ataxia (FA). 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., CEO 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.”

The vatiquinone New Drug Application (NDA) is based on data from the placebo-controlled MOVE-FA study, as well as results from two long-term studies involving pediatric and adult patients with fibromyalgia. These three studies provide substantial, enduring, and clinically significant evidence of slowing the progression of the disease in key areas. Moreover, these studies demonstrate that vatiquinone is safe and well-tolerated across all age groups studied.

In 2024, PTC submitted four NDA applications to the FDA, and all four were successfully accepted for review.