PTC Therapeutics Sets FDA Target Date For Sepiapterin NDA Review And Publishes Phase 3 Trial Results

(RTTNews) - PTC Therapeutics Inc. (PTCT) announced that the FDA has set a target action date of July 29, 2025, for the review of the New Drug Application (NDA) for sepiapterin. The NDA was submitted with an indication for the treatment of pediatric and adult patients with phenylketonuria (PKU), including all age groups and the full spectrum of disease subtypes.

Additionally, PTC revealed that results from the Phase 3 APHENITY trial were recently published in The Lancet, a leading peer-reviewed medical journal. The NDA includes findings from this pivotal trial, as well as data from the ongoing APHENITY open-label extension study, which highlights the long-term effectiveness of sepiapterin and its potential to allow patients to relax dietary restrictions while maintaining control over phenylalanine (Phe) levels.

Notably, the latest results from the Phe tolerance assessment indicate that around 60% of participants can consume protein levels exceeding the age-adjusted recommended daily allowance for individuals without PKU while keeping their Phe levels below 360 micromoles per liter.

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