PTC Therapeutics Inc. (PTCT) has announced that the U.S. Food and Drug Administration (FDA) has designated July 29, 2025, as the target date for the review of the New Drug Application (NDA) for sepiapterin. This application seeks approval for the drug's use in treating both pediatric and adult patients with phenylketonuria (PKU), extending across all age groups and encompassing the entire range of disease subtypes.
Furthermore, PTC has disclosed that the findings from the Phase 3 APHENITY trial have recently been published in "The Lancet," a highly regarded peer-reviewed medical journal. The NDA submission includes these pivotal trial results, along with data from the ongoing APHENITY open-label extension study. This data emphasizes sepiapterin's long-term efficacy and its potential role in enabling patients to relax dietary restrictions while still effectively managing phenylalanine (Phe) levels.
Importantly, recent results from the Phe tolerance assessment show that approximately 60% of participants are able to consume protein amounts that exceed the age-adjusted recommended daily allowance for individuals without PKU, while maintaining their Phe levels below 360 micromoles per liter.
