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Translarna (Ataluren): Disappointing news for the Duchenne community

Amsterdam, January 26, 2024-  The Duchenne community, represented by the World Duchenne Organization, was deeply disappointed to be informed about the recommendation of EMA’s human medicines committee (CHMP) in September 2023 not to renew the Conditional Marketing Authorisation for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy (DMD) with nonsense mutations, which received Conditional Marketing Authorisation (CMO) in Europe in 2014.

As stated in the company press release from 15 October 2023, the CHMP agreed to re-examine its decision in January this year based on CHMP procedural guidance.Today we are very disappointed and sad to report a negative outcome to the re-examination procedure, which means that Translarna will no longer be available in Europe.

Our hearts go out to all the boys and young men with DMD who have taken part in the clinical trials with Translarna and/or been on Translarna for many years, as well as those who were hoping to start this treatment in the near future. Many of them have seen considerable benefit over the years and are very concerned about what will happen when they no longer have access, particularly when there is no other drug available to treat the specific kind of mutation addressed by Translarna.

It is a sad day for the whole community worldwide. Even if the benefit is small in some cases, Translarna is a safe drug with little to no side-effects that was doing something to alleviate the course of the disease and for others meant longer ambulation, postponed need for ventilation and other benefits, as demonstrated by the RWD gathered in the STRIDE registry.

EMA shared the following information for patients and carers

  • A full review of all the data available on the benefits and risks of Translarna has concluded that the effectiveness of the medicine has not been confirmed in patients with nonsense mutation Duchenne muscular dystrophy.
  • The review considered results from studies, data from patient registries and the views of experts in neurology and patient representatives, as well as contributions from families, individual doctors, patients and healthcare professional organisations.
  • The review included the evaluation of a recent study involving patients with nonsense mutation Duchenne muscular dystrophy, which compared the effect of Translarna with that of placebo (a dummy treatment) after 18 months of treatment. The study failed to show that Translarna had a beneficial effect in those patients with a progressive decline in their ability to walk, although they were expected to benefit most from treatment with Translarna.
  • In addition, no conclusion on the benefits of Translarna could be drawn from a study based on data from patient registries information due to several issues and uncertainties with the data.
  • Therefore, EMA concluded that the benefits of Translarna have not been confirmed and has recommended that the marketing authorisation forTranslarna should not be renewed. Once this recommendation is confirmed by the European Commission, the medicine will no longer be authorised in the EU.
  • If you or your child is receiving Translarna, you should speak to your doctor about this decision and what it means for you or your child’s treatment.

For the full press release:

EMA confirms recommendation for non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna | European Medicines Agency (europa.eu)