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

EMA’s human medicines committee (CHMP) 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.

Because Translarna was meant to address an unmet medical need for a serious disease, it received a conditional marketing authorisation in July 2014. This type of authorisation allows a medicine to be authorised on the basis of less comprehensive (complete) data than are normally required, and when the benefits of having it available earlier outweigh any risks associated with using the medicine while waiting for further evidence. Medicines with a conditional marketing authorisation are subject to specific post-authorisation obligations that aim to generate comprehensive data…