Trial Profile

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

Status: Recruiting

Phase of Trial: Phase III

Latest Information Update: 26 Jun 2025

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At a glance

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

Most Recent Events

23 Jun 2025 According to a PTC Therapeutics media release, The Sephience European launch will be initiated in Germany in the first half of July.
23 Jun 2025 According to a PTC Therapeutics media release, Sephience (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The authorization includes a broad label inclusive of all ages and disease severities.
08 Oct 2024 Planned End Date changed from 7 Feb 2025 to 30 Apr 2026.

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