PTC Therapeutics Announces Sepiapterin NDA Submission to FDA

Core Insights - PTC Therapeutics has submitted a New Drug Application (NDA) for sepiapterin to the U.S. FDA, aimed at treating both pediatric and adult patients with phenylketonuria (PKU) [10] - The NDA is supported by significant clinical trial data, showing a mean reduction in phenylalanine (Phe) levels of 63% in the overall treated population and 69% in classical PKU patients [11] - The company is also pursuing marketing authorization in Europe, Japan, and Brazil, with applications expected later in 2024 [1][10] Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on developing and commercializing medicines for rare disorders, leveraging scientific expertise and a robust pipeline [4] - The company's mission is to provide access to innovative treatments for patients with limited options, aiming to maximize value for stakeholders [4] Product Details - Sepiapterin is an oral formulation that serves as a precursor to tetrahydrobiopterin, crucial for metabolic processes, and is more bioavailable than synthetic BH4 [2] - The clinical trial data indicates that 84% of subjects achieved Phe control below 360 µmol/L, and 22% normalized their Phe levels, suggesting potential for diet liberalization [11]