Update on the European Medicines Agency recommendation not to renew Translarna marketing
authorisation in the European Union
On Friday, 22 September, PTC Therapeutics, the company that manufactures Translarna, issued a
statement to Duchenne Patient Advocacy Organisations that provides an update on the European
Medicine Agency’s human medicines committee (CHMP) decision not to recommend renewal of the
conditional marketing authorization for Translarna.
The statement confirms that PTC will be appealing the decision through an established re-examination process and that the CHMP opinion does not cover Great Britain (England, Scotland and Wales). Therefore patients in England, Scotland and Wales are not in scope of the CHMP opinion.
The statement also emphasises that the CHMP did not express concerns regarding the safety of Trannslarna and that more than 3,000 patients have been treated with Translarna worldwide and more than 700 patients have been involved in the clinical trials.
Under current post-Brexit arrangements, Northern Ireland is within the scope of the CHMP opinion. Patients in Northern Ireland currently receiving Translarna will be able to remain on treatment and new patients will be able to start Translarna during the re-examination process.
According to CHMP guidelines, the re-examination procedure could last until January 2024, with
European Commission adoption within 67 days after that i.e. by the end March 2024.
We are monitoring the situation and will continue to keep the community informed of developments.
We are here to support you
We know many people in our community may feel concerned about this news. If you would like some more support or information, please contact us on info@actionduchenne.org or 07535498506.
Find out more about treatment updates, next steps in research and hear from the pharmaceutical companies themselves at our Annual International Conference. The 2 day conference will be held on the 10th and 11th November in Hinckley, Leicester and will bring together families, clinicians, therapists, researchers, and pharmaceutical companies from across the world. The theme for this year’s conference is ‘Educating, supporting, empowering’ focussing’ on key areas of Duchenne muscular dystrophy, enabling you to gain understanding, build your support network and take control.
Tickets are free for Duchenne families and those living with Duchenne, and there is a £100 grant available towards travel and accommodation.
