European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation
We are pleased to announce that the European Commission (EC) has decided not to adopt the Committee for Medicinal Products for Human Use's (CHMP) negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren).
The EC has asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a re-evaluation.
As a result, Translarna remains on the market and available for people in Ireland and other European countries consistent with its current marketing authorisation.
On 15 September 2023, the EMA's Scientific Committee for Medicinal Products for Human Use gave a negative opinion to the submission for the conversion of Translarna's conditional authorisation to full authorisation and the renewal of the existing conditional authorisation.
Following this decision many patient organisations (including MDI) and clinical networks throughout Europe mobilised together to express their support for maintaining Translarna™ (ataluren) as a medical option in the European Union. This included writing to national representatives on the CHMP and the launch of “Buy Some Time”. This was a campaign bringing together associations, families, and patients from all over Europe under one claim, together with a petition on Change.org that collected more than 15,000 signatures.
On 25 January 2024, the CHMP issued its second negative opinion for the renewal of the conditional marketing authorisation of Translarna™ (ataluren) following the review procedure.
The choice, therefore, was to appeal to the European Commission. Clinical networks for DMD, MDI and other patient associations throughout Europe wrote a letter to the European Commission and the EMA asking them to review the decision on Translarna™ (ataluren).
The EC announcement is a great victory for the international Duchenne and Becker community, who can continue using Ataluren!