PTC Therapeutics announced on April 25, 2025, that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion. This opinion is for the marketing authorization application for Sephience (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU).
The positive opinion includes a broad label, encompassing all ages and disease severities of PKU patients. This is a significant step towards bringing Sephience to the European market, addressing a substantial unmet medical need.
PTC expects the European Commission to ratify this marketing authorization in approximately two months, making the decision applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Launch planning in Europe is already underway, with Germany identified as a priority market for early access.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.