Sephience PTC Therapeutics International Limited · Authorised · authorised 19 Jun 2025 · ATC A16AX28
Therapeutic indication (EMA)
Treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU).
Active substance
Sepiapterin
- US FDA 1
- EMA 1
- Health Canada 2
US FDA United States — FDA approvals (Drugs@FDA)
| Product | Strength | Form · route | Applicant | Status | First approved |
|---|---|---|---|---|---|
| SEPHIENCE | 250MG/PACKET; 1GM/PACKET | powder · oral | PTC THERAP | Prescription | 28 Jul 2025 |
EMA European Union — centrally authorised medicines
Canada Canada — marketed products (Health Canada)
Values as published by each source: SFDA human drug list (4-2026, as published by the Council of Health Insurance; indications from the CHI Daman Drug Formulary), Drugs@FDA, EMA medicines data, Health Canada Drug Product Database (Open Government Licence – Canada), CMS NADAC, NHS Drug Tariff Part VIIIA (NHSBSA Copyright, Open Government Licence v3.0), WHO Model List of Essential Medicines (CC BY 3.0 IGO; regrouped by active substance, not created by WHO), EMA PRAC, US FDA safety-related labeling changes and MHRA Drug Safety Update (Open Government Licence v3.0). Sources and reuse. Not a substitute for the official registers or the product information.