On 15 September 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) decided against renewing the marketing authorisation for Translarna (ataluren) in Duchenne muscular dystrophy (DMD). Developed by the laboratory PTC Therapeutics, the drug has until now benefited from a conditional marketing authorisation granted by the EMA for patients with a ‘nonsense’ mutation of the DMD gene who can walk.
This opinion, announced by the EMA on January 26, is based on the results of the confirmatory phase III trial and those of a study comparing the data from the STRIDE register and those from the CINRG register, combined with all the data collected over the last 10 years. The CHMP experts consider the data to be complete and conclude that the benefit-risk ratio of Translarna is negative.
The European Commission is due to give its opinion within the next two months. Pending this decision, French patients will continue to have unchanged access to the treatment; but it is too early to know what operational arrangements will be applied in France following the decision.
EMA confirms recommendation for non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna. EMA, 26 January 2024 press release
CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna™ (ataluren) Following Re-examination Procedure. PTC Therapeutics, 25 January 2024 press release
