Trial Profile

A Phase 3 Study of PTC923 in Subjects With Phenylketonuria

Status: Completed

Phase of Trial: Phase III

Latest Information Update: 08 Jan 2026

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At a glance

22 Dec 2025 According to a PTC Therapeutics media release, Sephience (sepiapterin) has been approved by the Japanese Ministry of Health, Labor and Welfare (MHLW) for the treatment of children and adults living with phenylketonuria (PKU). The label includes individuals of all ages and the full spectrum of disease severity.
10 Dec 2025 According to a PTC Therapeutics media release, company announced that Health Canada has approved Sephience (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU).
28 Jul 2025 According to a PTC Therapeutics media release, based on data from Phase 3 APHENITY trial and APHENITY long-term extension study,the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria.

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