Sephience? (sepiapterin) Granted Marketing Authorization by the European Commission for the Treatment of Children and Adults Living with Phenylketonuria (PKU)

發(fā)布時間： 2025-06-27 閱讀：109次

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On June 24, 2025,  PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that Sephience? (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The European approval is based on the highly statistically significant results from the Phase 3 APHENITY trial as well as evidence of durable treatment effect and the ability of study participants to liberalize their diet in the APHENITY long-term extension study. A New Drug Application (NDA) for sepiapterin remains on schedule for its FDA target action date of July 29, 2025. 

Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Sephience acts as a dual pharmacological chaperone (sepiapterin and BH4 each with its own binding affinity to variant PAH), including PAH variants commonly found in PKU and known to be insensitive to BH4, to improve the activity of the defective PAH enzyme, achieving a high concentration of BH4 intracellularly. By enhancing the conformational stability of misfolded PAH enzyme and increasing the intracellular concentrations of BH4, Sephience is able to effectively reduce blood Phe levels.