PTC Therapeutics announced on June 23, 2025, that Sephience (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). This authorization includes a broad label, covering all ages and disease severities.
The European approval is based on highly statistically significant results from the Phase 3 APHENITY trial, along with evidence of durable treatment effect from the APHENITY long-term extension study. This data also demonstrated the ability of study participants to liberalize their diet.
The marketing authorization is applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. The European launch of Sephience will be initiated in Germany in the first half of July, positioning it as a potential new standard of care.
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