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A Phase 3 Study of PTC923 in Subjects With Phenylketonuria

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Trial Profile

A Phase 3 Study of PTC923 in Subjects With Phenylketonuria

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 13 May 2025

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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.

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