Sephience (sepiapterin)
- Region
- 🇪🇺 EU
- Active substance
- sepiapterin
- Company
- PTC Therapeutics International Limited
- Status
- Authorised
- Therapeutic area
- Phenylketonurias
- CHMP opinion
- Fri 25 Apr 2025
- EU approval
- Thu 19 Jun 2025
- Designations
- Orphan
- Application
- EMEA/H/C/006331
- ATC
- A16AX28
- Official record
- www.ema.europa.eu
Indication
Treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU).
Regulatory milestones
Sephience: PDUFA target action date (phenylketonuria (PKU))
Ptc Therapeutics, Inc. (PTCT); sepiapterin; NDA; status: set; "PTC Therapeutics Provides Corporate Update and Reports First Quarter 2025 Financial Results – Strong revenue performance of $190 million – – Positive CHMP opinion for Sephience ™ (sepiapterin) received in April 2025, NDA review remains on track for July 29, 2025 PDUFA date – – Global Sephience launch activities…"; filed 2025-05-06 · source
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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.