Translarna currently has a conditional marketing approval (authorisation) from the EMA, given in July 2014. A conditional approval allows a medicine to be made available based on less far reaching information/data than is normally required. This is used when the EMA assesses that the benefits of having the treatment available earlier are greater than any risks from using the medicine while waiting for more evidence/information to come through.
The EMA has now concluded that the further studies that have since been carried out do not show a clear benefit from Translarna, and they are therefore not renewing its approval for marketing.
Reactions to this news
The company that manufactures Translarna, PTC, has expressed surprise and ‘extreme disappointment’ at the announcement. It plans to submit a request for this to be looked at again by the EMA. While this is happening Translarna will still be available on the market across the EU and available to patients until the process to look at it again has been completed – this is likely to be in early 2024.
The World Duchenne Organisation has expressed ‘deep disappointment’ at this news and has highlighted the importance of real-world data – information from patient surveys, patient health records etc. – being considered by regulators, particularly when considering clinical trials in complex conditions such as Duchenne. They highlight that in rare disease, it is not always possible to show substantial benefit within the timeframe of clinical trials.
Read the World Duchenne Organisation's statement.
What this means for people in the UK
In the UK, Translarna has a marketing authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA).
In January 2023, the National Institute of Health and Care Excellence (NICE) published final guidance recommending Translarna as an option for treating Duchenne muscular dystrophy resulting from a 'nonsense mutation’ in the dystrophin gene in people two years and over who can walk.
We have been leading the fight for access to the treatment since 2016, working alongside families, clinicians, and other patient groups.
In its guidance (which applies to England, Wales and Northern Ireland), NICE confirmed that Translarna is clinically effective and that it is likely to slow the progression of Duchenne muscular dystrophy for eligible patients. It also recognised that it has a positive impact on both the lives of people receiving it and on caregivers. Translarna is available in Scotland through the ‘ultra-orphan pathway’ and the Scottish Medicines Consortium (SMC) is due to appraise it in 2025.
Read our update concerning PTC's statement (released on 22 September 2023).