PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA

Core Viewpoint - PTC Therapeutics announced that the FDA issued a Complete Response Letter (CRL) for the New Drug Application (NDA) of vatiquinone, indicating that substantial evidence of efficacy was not demonstrated and an additional study is required for resubmission [1][2]. Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on developing and commercializing medicines for rare disorders, with a commitment to providing access to best-in-class treatments [5]. Product Information - Vatiquinone is a first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), aimed at addressing mitochondrial dysfunction and oxidative stress in Friedreich's ataxia, with clinical studies showing potential impacts on mortality risk and various neurological symptoms [3]. Disease Context - Friedreich's ataxia is a rare neuromuscular disorder caused by a genetic defect in the frataxin gene, leading to debilitating symptoms and affecting approximately 25,000 people globally [4].