Biopharmaceutical company PTC Therapeutics Inc (NASDAQ: PTCT) announced on Monday that the US Food and Drug Administration (FDA) has approved SEPHIENCE (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU).

This approval includes broad labelling for the treatment of hyperphenylalaninemia (HPA) in adult and paediatric patients 1 month of age and older with sepiapterin-responsive PKU.

PKU is a rare, inherited metabolic disease, which affects the brain.

The FDA approval is based on 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 was recently granted marketing authorisation by the European Commission. Review of approval applications is ongoing in several other countries including Japan and Brazil.