PTC Therapeutics Announces European Approval for Sephience™ in PKU Treatment

The European Commission's approval of Sephience™ encompasses a broad label, allowing the treatment to be used by both children and adults across all ages and varying severities of PKU. This comprehensive indication enhances the potential of Sephience to become a standard of care for patients suffering from this inherited disorder.

Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, expressed enthusiasm about the approval, stating, "The European approval of Sephience is a great step in our efforts to bring this safe and highly effective therapy to children and adults affected by PKU worldwide." Dr. Klein emphasized that the broad label supports the treatment's potential to address all key patient segments within the PKU community.

The marketing authorization is underpinned by the results from the Phase 3 APHENITY trial, which demonstrated highly statistically significant outcomes. Moreover, evidence from the long-term extension study showed that participants could liberalize their diets, indicating a durable treatment effect of Sephience. This clinical validation is crucial in establishing the efficacy and safety of sepiapterin as a therapeutic option for PKU patients.

The launch of Sephience in Europe is set to initiate in Germany during the first half of July 2025, providing timely access to this novel treatment for patients in need. Furthermore, PTC Therapeutics is progressing with a New Drug Application (NDA) for sepiapterin in the United States, which is scheduled for review by the FDA with a target action date of July 29, 2025. In addition, regulatory reviews are ongoing in other countries, including Japan and Brazil, indicating PTC's commitment to global commercialization of Sephience.

Phenylketonuria (PKU) is a rare genetic disorder caused by a defect in the gene responsible for producing the enzyme needed to metabolize phenylalanine (Phe), an essential amino acid. If left untreated, elevated levels of Phe can lead to severe nerve damage, intellectual disabilities, and other significant health issues. PKU is typically diagnosed through newborn screening, affecting an estimated 58,000 individuals worldwide.

PTC Therapeutics is a global biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative medicines aimed at addressing unmet medical needs in rare disorders. The company leverages its scientific expertise and commercial infrastructure to maximize the value of its products for patients and stakeholders alike.

As the European launch of Sephience approaches, PTC Therapeutics stands poised to make a meaningful impact in the lives of those affected by PKU, offering hope for improved health outcomes and quality of life.

For further updates on PTC Therapeutics and its innovative therapies, interested parties can follow the company on their social media platforms or visit their website.