Sephience™ Receives EU Approval for Treating PKU in All Ages

Sephience™ Granted Marketing Authorization for PKU Treatment



PTC Therapeutics, Inc. (NASDAQ: PTCT) recently celebrated the significant milestone of receiving marketing authorization for Sephience™ (sepiapterin) from the European Commission. This approval allows for the treatment of both children and adults affected by phenylketonuria (PKU), a rare inherited metabolic disorder.

A Major Step Forward



According to Matthew B. Klein, M.D., the CEO of PTC Therapeutics, this regulatory achievement is more than just a corporate success; it represents a new hope for patients worldwide suffering from PKU. The approved treatment showcases a broad therapeutic range, as it encompasses patients of all ages and varying degrees of disease severity, thus underscoring its comprehensive applicability. The marketing authorization was founded on the notable outcomes observed during the Phase 3 APHENITY trial. These trials highlighted a statistically significant reduction in blood phenylalanine (Phe) levels, allowing patients to enjoy a more liberalized diet.

Implications of Approval



This marketing authorization is crucial as it is applicable across all 27 EU member states, alongside Iceland, Norway, and Liechtenstein. The initial launch of Sephience will take place in Germany, expected to begin in the first half of July. This represents a first critical step in what the company hopes will be a broader rollout across Europe, providing essential treatment options to potentially 58,000 patients living with PKU globally.

In addition to its European success, a New Drug Application (NDA) has been submitted to the FDA with a target action date set for July 29, 2025, indicating that enthusiasm is also building in the U.S. market. The company is also moving forward with approval applications in other countries like Japan and Brazil.

Understanding Phenylketonuria (PKU)



PKU is an inherited metabolic disorder characterized by the inability to properly break down phenylalanine, an amino acid found in many protein-rich foods. Left untreated, elevated levels of Phe can lead to severe cognitive impairments, seizures, and various emotional disturbances. This makes early diagnosis—typically carried out through newborn screening—critical to managing the condition effectively.

For many patients, symptom management requires lifelong dietary restrictions; however, Sephience acts as a dual pharmacological chaperone that aids in enhancing the activity of the defective phenylalanine hydroxylase (PAH) enzyme. This is crucial for managing blood Phe levels effectively, which can significantly improve the quality of life for those affected.

PTC Therapeutics' Commitment



PTC Therapeutics is committed to developing clinically differentiated medicines aimed at rare disorders. The company views itself as a patient-centered organization, focusing on delivering best-in-class treatments to individuals with unmet medical needs. The solid scientific foundation combined with a robust global commercial infrastructure drives its vision for future growth, making it a key player in the biopharmaceutical landscape.

For more information on PTC Therapeutics and its range of products, visit PTC Bio's website. Stay updated on exciting developments by following them on Facebook, Twitter, and LinkedIn.

As Sephience represents a pioneering step in PKU management, it highlights a significant advancement in metabolic disorder treatments, reiterating the importance of ongoing research and innovation in this challenging field.

Topics Health)

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