EMA has recommended not to renew the marketing authorisation for Translarna in the EU
It has been announced today that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended that it not renew the conditional marketing authorisation for Translarna (also known as ataluren).
Translarna is a medicine for treating patients with Duchenne muscular dystrophy (DMD) that is taken orally.
This announcement will only affect patients in the European Union. The Medicines and Healthcare products Regulatory Agency (MHRA) is the relevant regulatory body in the UK, and the CHMP’s recommendation does not directly inform their decision-making.
Conditional Market Authorisation
Translarna was developed by PTC Therapeutics, and received conditional market authorisation (CMO) 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.
Why the recommendation has been made
The recommendation follows 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.
What happens next
According to the PTC Therapeutics, Translarna will remain on the market and available to patients with DMD in the EU until the re-examination process is completed. This is expected in early 2024.
This means that, in the EU, no new patients will be treated with Translarna. Those on it will continue to be treated with it until at least the start of 2024, while PTC Therapeutics appeals the decision at the re-examination. Availability in the EU after the re-examination will depend on its outcome.
In the UK, access will continue for patients as normal, unless the MHRA chooses to review Translarna’s licence due to the EMA’s decision. If that happens, we will update the community.
Importance of treatment options
This is distressing news for patients in the EU who are being treated with Translarna, and those hoping to start it in the near future.
We will be in contact with PTC Therapeutics and the MHRA about what this means for DMD patients in the UK, and will post an update when we know more.