PTC Therapeutics has announced that the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a negative recommendation on June 28th regarding renewing Translarna’s marketing authorization for treating patients with Duchenne muscular dystrophy caused by a nonsense mutation. This follows the European Commission’s (EC) request in May for a fresh review, which we previously reported.
PTC Therapeutics have stated their plans to challenge the negative recommendation by requesting a re-examination of the opinion. In their announcement, PTC states the marketing authorization for Translarna remains in effect, pending the outcome of the re-examination procedure and subsequent EC ratification. Even if the negative opinion stands, PTC estimates Translarna will likely stay available until the end of 2024.
We are disappointed that the CHMP has maintained its negative opinion on Translarna despite the request of the European Commission to consider the totality of evidence including the STRIDE real world evidence in a revised opinion. The Scientific Advisory Group convened as part of this most recent review procedure concluded that the evidence of significant efficacy provided by STRIDE ‘should not be ignored.’ This view of the CHMP expert committee is consistent with the view of DMD key opinion leaders worldwide. We will request re-examination of the CHMP opinion to ensure that we make every effort to maintain availability of Translarna on the market for as long as possible for boys and young men with nmDMD.”
Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics
Following the UK leaving the European Union, decisions made by the EMA do not impact the availability of Translarna in the UK. The Medicines and Healthcare products Regulatory Agency (MHRA) and National Institute for Health and Care Excellence (NICE) are responsible for the review of treatments in the UK. The 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 in February 2023.
The MHRA are currently conducting their independent review of Translarna, and Action Duchenne remains committed to engaging in this review process as patient experts to ensure we are representing your views and experiences.
If you would like to read more about the decision and PTC therapeutics response, please follow the link below.
