PTC Therapeutics Says Health Canada Approves Sephience To Treat Phenylketonuria In Children, Adults

(RTTNews) - Biopharmaceutical company PTC Therapeutics Canada ULC announced Wednesday 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.