In September 2023 we shared news that the European Medicine Agency’s (EMA) human medicines committee (CHMP) had released a recommendation not to renew the approval (authorisation) for the marketing of Translarna – this is the approval for Translarna to be made available in the European Union.
The company that manufactures the treatment, PTC, appealed the decision and requested a formal re-examination. This process is now finished and on Friday 26 January 2024 the EMA confirmed its recommendation for non-renewal of the marketing authorisation. The process found that while there was evidence of benefit from the treatment, it’s not statistically significant. There are no concerns about the safety of the treatment.
This decision means that Translarna will shortly stop being available to patients in the EU. The decision does not affect availability in England, Wales and Scotland as marketing authorisation in Great Britain is the responsibility of the Medicines and Healthcare products Regulatory Agency (MHRA) and not the EMA.
In Northern Ireland, which is currently covered by the EMA, a system is in place that is designed to ensure that people in Northern Ireland can continue to access treatments available in Great Britain where there is a risk that clinical need for that treatment cannot be met. This is called the Northern Ireland MHRA Authorised Route (NIMAR) and we understand that its application is being explored in this case.
We are seeking clarification on availability in Northern Ireland.
A year ago, 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. In its guidance (which applies to England, Wales and Northern Ireland), NICE confirmed that Translarna is clinically effective and that it’s 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. We are therefore fully supportive of the treatment’s continued availability and are disappointed by the outcome of the EMA process.
Translarna is available in Scotland through the ‘ultra-orphan pathway’ and the Scottish Medicines Consortium (SMC) is due to appraise it in 2025.
We’ll continue to keep you up to date as we receive further information.
If you would like to talk to someone, our helpline are here to offer free information and support. We’re open Monday – Thursday 10am-2pm. Call 0800 652 6352 or email info@musculardystrophyuk.org.