PTC Therapeutics announced on July 28, 2025, that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). This approval includes broad labeling for hyperphenylalaninemia (HPA) in patients 1 month of age and older with sepiapterin-responsive PKU.
SEPHIENCE is indicated for use in conjunction with a phenylalanine (Phe)-restricted diet. The FDA approval is based on significant efficacy and safety evidence from the Phase 3 APHENITY trial, as well as the durability of treatment effect observed in the APHENITY long-term extension study.
This approval marks an important milestone for the PKU community, offering a new treatment option that has the potential to improve the quality of life for individuals of all ages and PKU subtypes. SEPHIENCE was also recently granted marketing authorization by the European Commission.
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