🇪🇺 PTC923 in European Union

EMA authorised PTC923 on 19 June 2025

Marketing authorisation

EMA — authorised 19 June 2025

  • Application: EMEA/H/C/006331
  • Marketing authorisation holder: PTC Therapeutics International Limited
  • Local brand name: Sephience
  • Indication: Treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU).
  • Pathway: orphan
  • Status: approved

The European Medicines Agency (EMA) has granted marketing authorisation for Sephience (PTC923) to treat hyperphenylalaninaemia in adult and paediatric patients with phenylketonuria (PKU). This authorisation was granted under the orphan designation pathway. Sephience is the local brand name for PTC923, developed by PTC Therapeutics International Limited.

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PTC923 in other countries

Same drug, different regulators. Each page below lists the local marketing authorisation, HTA position, pricing, and adverse-event data.

Frequently asked questions

Is PTC923 approved in European Union?

Yes. EMA authorised it on 19 June 2025.

Who is the marketing authorisation holder for PTC923 in European Union?

PTC Therapeutics International Limited holds the EU marketing authorisation.