PTC Therapeutics(PTCT) Prnewswire·2025-02-19 12:00Core Points - PTC Therapeutics has announced that the FDA accepted the New Drug Application (NDA) for vatiquinone, targeting a PDUFA action date of August 19, 2025, for the treatment of Friedreich's ataxia [1][2] - Vatiquinone, if approved, would be the first therapy for pediatric patients with Friedreich's ataxia and aims to provide a safe and effective treatment alternative for adults [2] - The NDA is based on data from the placebo-controlled MOVE-FA study and two long-term studies, demonstrating significant evidence of slowing disease progression and safety across all age groups [2][5] Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on discovering, developing, and commercializing medicines for rare disorders [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, targeting pathways disrupted in Friedreich's ataxia, and has shown potential in alleviating mitochondrial dysfunction and oxidative stress [4] - The MOVE-FA trial enrolled 146 patients, primarily under 18 years, and while the primary endpoint did not reach statistical significance, significant effects were observed on specific subscales [5] Disease Context - Friedreich's ataxia is a rare, debilitating neuromuscular disorder affecting approximately 25,000 people globally, characterized by progressive loss of coordination and muscle strength [6]