European Commission Sends Translarna™ Decision Back to CHMP for Re-evaluation

20 May 2024

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).

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 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, available at PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation | PTC Therapeutics, Inc. (ptcbio.com)

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. Read the full NICE guidance.