A Phase 3 Study of PTC923 in Subjects With Phenylketonuria
Latest Information Update: 13 May 2025
At a glance
- Drugs Sepiapterin (Primary)
- Indications Phenylketonuria
- Focus Registrational; Therapeutic Use
- Acronyms APHENITY
- Sponsors PTC Therapeutics
Most Recent Events
- 25 Apr 2025 According to a PTC Therapeutics media release, the New Drug Application (NDA) for sepiapterin is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of July 29, 2025.
- 25 Apr 2025 According to a PTC Therapeutics media release, company announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU).
- 20 Mar 2025 According to a PTC Therapeutics media release, company presented new data from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting.