- PTC Therapeutics (PTCT, Financial) receives positive CHMP opinion for Sephience™ in Europe.
- Sephience set to treat all ages and severities of PKU, with market launch imminent.
- FDA review underway, decision expected by July 29, 2025; approvals also sought in Japan and Brazil.
PTC Therapeutics, Inc. (PTCT) announced receiving a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency for Sephience™ (sepiapterin). This recommendation supports the drug’s use in treating phenylketonuria (PKU) across all age groups and disease severities, marking a significant step toward addressing unmet medical needs in this patient population.
The company plans to launch Sephience in Europe immediately following the European Commission's expected ratification in about two months. This authorization will cover all 27 European Union member states, plus Iceland, Norway, and Liechtenstein, with an initial focus on the German market and other key European markets where named patient access will be available promptly.
Sephience, a synthetic formulation designed to lower phenylalanine levels, also has approval applications under review by the U.S. Food and Drug Administration (FDA), with a target action date of July 29, 2025. Additionally, regulatory submissions are ongoing in Japan and Brazil. PTC's global regulatory strategy demonstrates a comprehensive approach to making Sephience available to PKU patients worldwide, addressing a condition that profoundly impacts quality of life through strict dietary restrictions.
