EMA re-confirms non-renewal of conditional marketing authorisation of Duchenne muscular dystrophy medicine, Translarna
Following a re-examination of available data, EMA’s human medicines committee (CHMP) has confirmed its previous recommendation to not renew the conditional marketing authorisation for Translarna (ataluren). This last round of assessment concluded that the effectiveness of Translarna has not been confirmed.
Translarna is used for treating patients with Duchenne muscular dystrophy aged 2 years and older who are able to walk and whose disease is caused by a type of genetic defect called a ‘nonsense mutation’ in the dystrophin gene.
After this assessment the CHMP recommendation remained negative.
The company then requested another re-examination, and the CHMP re-assessed the results from a study carried out after authorisation as a specific obligation (study 041) as well as patient registries data, taking into account new analyses provided by the company.
EMA will now send the CHMP’s opinion to the European Commission, which will issue a final legally binding decision applicable in all EU Member States.
The CHMP adopted a new opinion on 27 June 2024. The company asked for re-examination of this latest CHMP’s opinion on 11 July 2024.
The CHMP has now re-examined its opinion, and its recommendation will be sent to the European Commission for a legally binding decision applicable in all EU Member States.