European Medicines Agency (EMA) recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna

15 September 2023 – EMA’s human medicines committee has recommended not renewing the marketing authorisation for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy whose disease is caused by a type of genetic defect called a ‘nonsense mutation’ in the dystrophin gene and who are able to walk. The recommendation follows the full re-evaluation of the benefits and risks of the medicine during the renewal of its marketing authorisation, including results of a new study which failed to confirm Translarna's effectiveness.

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