PTC Therapeutics, Inc. has announced that the US Food and Drug Administration (FDA) has approved SEPHIENCE (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU).

The approval includes broad labelling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU, the company said in a statement.

Matthew B. Klein, Chief Executive Officer of PTC Therapeutics, said, “The broad labelling reflects the potential of Sephience to meet the significant unmet need of PKU patients. The Sephience clinical data, along with our expertise in launching rare disease therapies, position Sephience to become the future standard of care. Our experienced customer-facing teams are ready to bring this therapy to children and adults with PKU in the United States as quickly as possible.”

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

“The approval marks an exciting milestone for the PKU community,” said Catherine Warren, Executive Director of the National PKU Alliance. “This progress brings renewed hope, and we are eager to see the positive impact this new treatment option will have on advancing care and potentially improving quality of life for individuals of all ages and PKU subtypes that respond to this therapy.”

SEPHIENCE was recently granted marketing authorisation by the European Commission. Review of approval applications is ongoing in several other countries, including Japan and Brazil, the company stated.