PTC Therapeutics has announced today that the European Commission (EC) has decided not to adopt the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) negative opinion of January 24, 2024 on the annual renewal of the conditional marketing authorisation of Translarna™ (ataluren). Instead the EC has returned the opinion to the CHMP for re-evaluation. As a result, Translarna remains currently available in the European Union for eligible patients. The EC has asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion.
For more information, please refer to the PTC Therapeutics press release.
Following Brexit, the EMA does not have authority over UK marketing authorisation. This is the responsibility of the Medicines and Healthcare products Regulatory Agency (MHRA). The MHRA has begun its own review of Translarna and we are seeking clarification as to whether the EC’s decision will impact this process. We will keep the community informed as soon as more information becomes available.
In February 2023, NICE recommended Translarna as a treatment for children aged two and over with Duchenne muscular dystrophy, making it available on the NHS in England, Wales and Northern Ireland. We acted as a patient expert in the appraisal process, presenting the views of the Duchenne community after extensive consultation and engagement.
Would you like to know more about Duchenne muscular dystrophy? Increase your knowledge and understanding of Duchenne with our bite-sized science video series.
Section 1 – Facts about Duchenne muscular dystrophy
Section 2 – Signs and Symptoms of Duchenne muscular dystrophy
Section 3 – Diagnosis of Duchenne muscular dystrophy
Section 4 – Crucial Genetic Terminology
Section 5 – Genetics – Blueprint of Duchenne muscular dystrophy
Action Duchenne Annual International Conference 2024, 8th and 9th November: REGISTER YOUR INTEREST NOW!
