Access to treatments

European Commission rejects European Medicines Agency’s recommendation not to renew Translarna

The European Commission has rejected the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommendation that it not renew the conditional marketing authorisation for Translarna. The European Commission has sent the case back to the CHMP for another re-evaluation.

Review of Translarna

Translarna (also known as ataluren) is a medicine for treating patients with Duchenne muscular dystrophy (DMD). CHMP made the recommendation following a full re-evaluation by CHMP of the benefits and risks of Translarna during the renewal of its marketing authorisation, including results of a new study which failed to confirm Translarna’s effectiveness.

This rejection of the decision by the European Commission means that the current marketing authorisation remains in the European Union, allowing all eligible patients continued access during this re-evaluation process.

Conditional Market Authorisation

Translarna was developed by PTC Therapeutics, and received conditional market authorisation in Europe in 2014 for people with DMD who are aged two years and older and ambulatory (able to walk). It has been available to eligible patients in the UK since then.

It was granted this authorisation because of the unmet medical need for DMD. This type of authorisation allows a medicine to be authorised based on less complete data than is 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.

MHRA is reviewing Translarna

The Medicines and Healthcare products Regulatory Agency (MHRA) is the relevant regulatory body in the UK, and it is currently reviewing Translarna’s licence. A decision is expected by the end of the year.

Published on 23 May 2024

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