PTC Therapeutics announced on February 19, 2025, that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for vatiquinone. The application, which seeks approval for the treatment of children and adults living with Friedreich's ataxia (FA), has been granted Priority Review.
A Prescription Drug User Fee Act (PDUFA) target action date of August 19, 2025, has been assigned for the vatiquinone NDA. This priority review status reflects the significant unmet medical need for patients with FA, particularly for pediatric patients for whom there are currently no approved therapies.
The vatiquinone NDA is supported by data from the placebo-controlled MOVE-FA study and two long-term studies, which collectively demonstrated significant, durable, and clinically meaningful evidence of slowing disease progression. These studies also showed that vatiquinone is safe and well-tolerated across all age groups.
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