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PTC Therapeutics: Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna (Ataluren) in Europe

January 31, 2024

Translarna™ (ataluren) will be withdrawn as a therapy for Duchenne muscular dystrophy patients with nonsense mutations in Europe. The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion following the re-examination procedure for conditional marketing authorization. This decision comes after the CHMP agreed to re-examine the therapy’s status in January 2024. Defeat Duchenne Canada is disappointed by this recommendation, as Translarna has provided significant benefits to many people living with Duchenne in Europe.

“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.”

World Duchenne Organization

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 organizations.
  • 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 for Translarna should not be renewed. Once this recommendation is confirmed by the European Commission, the medicine will no longer be authorized 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.