US pharmaceutical company PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on Monday that it has received marketing authorisation from the European Commission for Sephience (sepiapterin) to treat both children and adults with phenylketonuria (PKU), including a broad label that spans all ages and severities of the disease.

The decision was supported by statistically significant data from the Phase 3 APHENITY trial and its long-term extension, which showed sustained treatment effects and dietary liberalisation in patients. The approval applies across all 27 European Union member states, Iceland, Norway, and Liechtenstein, with the initial product launch set for Germany in early July.

A New Drug Application for sepiapterin is currently under review by the US Food and Drug Administration, with a target action date set for 29 July 2025. Regulatory reviews are also underway in countries including Japan and Brazil.

Phenylketonuria is a rare genetic disorder that impairs the body's ability to break down phenylalanine, leading to severe and irreversible neurological damage if untreated. There are approximately 58,000 individuals living with PKU worldwide.

PTC Therapeutics views the broad label as indicative of the potential of Sephience to address the full PKU patient spectrum and become the new standard of care.