Translarna, a treatment for Duchenne muscular dystrophy, remains available in the UK

7 April 2025

Although Translarna (ataluren) will continue to be available in the UK, the European Commission has decided not to renew its market authorisation, meaning it will no longer be available in the EU.

Conditional approval

Translarna was first approved as a treatment for Duchenne muscular dystrophy (DMD) by the European Medicines Agency (EMA) in 2014. The EMA decides if a treatment should be approved for use in European Union countries. At the time this included the UK as it was before Brexit.

As there were some uncertainties over how well Translarna works, the EMA granted a conditional approval. They asked for further data to show the treatment works, before reassessing if the treatment should be approved.

EMA decision

In September 2023 the European Medicine Agency’s (EMA) human medicines committee (CHMP) recommended against the renewal of the approval of Translarna. This recommendation was passed to the European Commission (EC) who make a final legally binding decision.

PTC, the company that manufactures the treatment, appealed the decision and the EC asked the EMA to look at the data again. The EMA looked at the available data but continued to recommend against the renewal in January 2024, June 2024, October 2024 and March 2025.  

The EC has now accepted this recommendation and the approval (marketing authorisation) of Translarna will not be renewed. This decision means Translarna will no longer be available to people with DMD in the EU.

What does this mean for the UK?

Translarna remains available in the UK – England, Wales, Scotland and Northern Ireland. Since the UK is no longer part of the EU following Brexit, decisions about treatment approvals are now made by the Medicines and Healthcare products Regulatory Agency (MHRA).