Trial Profile

A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria

Status: Recruiting

Phase of Trial: Phase III

Latest Information Update: 16 May 2025

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At a glance

Drugs Sepiapterin (Primary)
Indications Phenylketonuria
Focus Adverse reactions; Therapeutic Use
Sponsors PTC Therapeutics

Most Recent Events

08 Oct 2024 Planned End Date changed from 7 Feb 2025 to 30 Apr 2026.
08 Oct 2024 Planned primary completion date changed from 7 Feb 2025 to 30 Apr 2026.
01 Oct 2024 According to a PTC Therapeutics media release, based on results form this and APHENITY trial, the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria (PKU). A Prescription Drug User Fee Act (PDUFA) target action date is expected to be provided in the Day 74 Letter.

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