EMA’s negative decision on Translarna upheld
This month (Oct 2024), the European Medicine Agency (EMA) finalised its review of Translarna (ataluren), and concluded that the medicine’s effectiveness for treating Duchenne muscular dystrophy (DMD) is not confirmed.
Translarna was first approved for DMD by the EMA in July 2014. Because there was uncertainty over the efficacy of the medicine at the time, it was given a conditional marketing authorisation and the company that manufactured it – PTC Therapeutics, Inc. – was asked to do more research to demonstrate Translarna’s benefits.
The EMA reviewed the available information, and reached an initial opinion to not renew the conditional marketing authorisation in September 2023. It gave this recommendation to the European Commission (EC), who could then make a legally binding decision applicable to all European Union member states. PTC appealed the recommendation, and the EC asked the EMA to think again.
Since then, the EMA has revisited the data and looked at new studies, but maintained its negative opinion in January 2024, June 2024, and now most recently in October 2024.
Unless there is another appeal, the EC could confirm the recommendation before the end of the year, meaning Translarna would no longer be an authorised in the EU.
This decision does not directly impact the UK. While Translarna’s authorisation was granted in 2014 when the UK was still a member of the EU, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has overseen giving medicines licencing since 2021. However there could ba an in-direct imapct; the MHRA is currently reviewing Translarna’s licence, and if the EU revokes access to Translarna it makes it more likely the MHRA will do the same.
To find out more about the EMA’s decision, the evidence they considered, and the reason for their negative opinion, click here.