EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna

EMA’s human medicines committee (CHMP) has recommended not renewing the conditional marketing authorisation for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy. Translarna is used in patients 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 CHMP issued an initial negative opinion on the renewal of the marketing authorisation for Translarna in September 2023, which was confirmed in January 2024 following a re-examination requested by the company marketing the medicine. Both rounds of assessment concluded that the effectiveness of Translarna had not been confirmed following a re-evaluation of the medicine’s benefits and risks.

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