Sephience approved for PKU

Sephience significantly lowers phenylalanine levels in PKU, as shown in a double-blind, randomized, placebo-controlled clinical study

PTC Therapeutics has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience (sepiapterin). The recommendation supports use in children and adults with phenylketonuria (PKU), across all ages and disease severities. “This positive opinion is great news for the PKU patient community in Europe for whom there remains a significant unmet medical need,” said Matthew B. Klein, Chief Executive Officer of PTC Therapeutics.

What is Sephience and what condition is it intended to treat?

Sephience (sepiapterin) is a treatment developed by PTC Therapeutics for phenylketonuria (PKU), a rare metabolic disorder affecting both children and adults.

What decision has the CHMP made regarding Sephience?

The CHMP has issued a positive opinion on the marketing authorization application for Sephience, recommending its use across all age groups and severities of PKU.

What clinical evidence supports the use of Sephience?

A double-blind, randomized, placebo-controlled clinical study showed that Sephience significantly reduced blood phenylalanine levels after 6 weeks compared with placebo.

What are the next steps for Sephience in Europe?

PTC Therapeutics plans to launch Sephience in Germany and other key European markets, with named patient access available immediately. The European Commission is expected to ratify the decision in approximately 2 months.

What is the regulatory status of Sephience outside Europe?

The New Drug Application for Sephience is under review by the FDA, with a target action date of July 29, 2025. Regulatory reviews are also ongoing in Japan and Brazil.