PTC Therapeutics Canada Says Health Canada Approves Sephience For Treatment Of Children And Adults Living With Phenylketonuria

- Broad label includes all age groups and full range of PKU subtypes -

TORONTO, Dec. 10, 2025 /CNW/ - PTC Therapeutics Canada ULC announced that Health Canada has approved Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month old and older with sepiapterin-responsive PKU.

The Health Canada approval is based on the evidence of significant efficacy and safety from the Phase 3 APHENITY trial as well as durability of treatment effect in the APHENITY long-term extension study.

Sephience is now commercially available in Canada. Sephience was recently approved by the U.S. Food and Drug Administration and granted marketing authorization by the European Commission.