PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive of all ages and disease severities.
“This positive opinion is great news for the PKU patient community in Europe for whom there remains a significant unmet medical need,” said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. “We look forward to initiating the European launch immediately following the adoption of this positive opinion by the European Commission. The data collected to date continue to support the potential for Sephience to provide a safe and effective treatment that not only lowers phenylalanine levels but also enables diet liberalization even for those with the most severe disease subtypes.”
Launch planning in Europe is well underway with priorities in Germany and other key European markets where named patient access will be immediately available.
PTC expects the European Commission to ratify the marketing authorization for Sephience in approximately two months. The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.
The New Drug Application (NDA) for sepiapterin is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of July 29, 2025. Review of approval applications is ongoing in several other countries including Japan and Brazil.
About Sephience™ (sepiapterin)
Sephience, an oral formulation of synthetic sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase (PAH) enzyme. First, Sephience is a precursor compound that is rapidly absorbed and converted intracellularly to tetrahydrobiopterin (BH4), a critical cofactor of PAH. Sephience also has an independent pharmacological chaperone effect, correcting PAH misfolding to enhance the enzyme function. Through this dual mechanism of action, Sephience effectively reduces blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients.
