- Broad labeling inclusive of all disease subtypes for individuals 1 month of age and older -

- PTC will host a conference call on Monday, July 28th at 5:00 pm ET -

WARREN, N.J., July 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the U.S. Food and Drug Administration (FDA) 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 of age and older with sepiapterin-responsive PKU.

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