PTC(PTC) Prnewswire·2025-02-19 12:00Core Insights - PTC Therapeutics has received FDA acceptance for the New Drug Application (NDA) for vatiquinone, targeting a PDUFA action date of August 19, 2025, for the treatment of Friedreich's ataxia (FA) [1][2] - Vatiquinone, if approved, would be the first therapy for pediatric patients with FA and a potential treatment alternative for adults, addressing a significant unmet need [2][4] - The NDA is based on data from the MOVE-FA study and two long-term studies, showing significant evidence of slowing disease progression and demonstrating safety across all age groups [2][5] Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on developing and commercializing medicines for rare disorders, with a robust and diversified pipeline [7] - The company has submitted four approval applications to the FDA in 2024, all of which have been accepted for review [3] Product Information - Vatiquinone is a first-in-class selective inhibitor of 15-Lipoxygenase, aimed at alleviating mitochondrial dysfunction and oxidative stress associated with Friedreich's ataxia [4] - The MOVE-FA trial enrolled 146 patients, primarily under 18 years, and while the primary endpoint was not statistically significant, a significant effect was observed on the upright stability subscale [5] Disease Context - Friedreich's ataxia is a rare, debilitating neuromuscular disorder affecting coordination and muscle strength, primarily caused by a genetic defect in the frataxin gene [6] - Approximately 25,000 individuals are affected by Friedreich's ataxia globally, with symptoms including poor balance, difficulty in speech and swallowing, and serious heart conditions [6]