Last month, EMA restarted the evaluation of an application to renew the conditional marketing authorisation for Translarna (ataluren), a medicine authorised for the treatment of Duchenne muscular dystrophy.

In January 2024, following a re-examination, EMA’s human medicines committee (the CHMP) had recommended not renewing the marketing authorisation for the medicine, based on its evaluation of the available data. This recommendation was then forwarded to the European Commission for an EU-wide final decision on the medicine’s authorisation.

The European Commission has now asked the CHMP to further consider whether the data available on Translarna are sufficiently comprehensive to conclude on the medicine’s benefit-risk balance. The CHMP has also been asked to consider whether additional real-world data (health data collected in routine care settings) brought to the attention of the Commission during its decision-making process may change the negative outcome previously reached by the Committee.

In addition, following the appellate judgment of the Court of Justice of the European Union about a different medicine, EMA has decided to convene a new scientific advisory group (SAG) on neurology for Translarna. This means that the evaluation will re-start from that stage of the initial renewal procedure. A scientific advisory group is comprised of scientific experts and is called upon to respond to specific questions posed by EMA’s committees during the evaluation of a medicine.  

A revised CHMP recommendation on the renewal of Translarna’s marketing authorisation is expected in the next few months. While EMA’s assessment is ongoing, the marketing authorisation for Translarna remains valid in the EU. Translarna’s availability in the different EU countries will depend on country-specific factors. If you have any questions, please speak with your doctor or contact your national competent authority.