Translarna (ataluren), developed by the laboratory PTC Therapeutics, has until now benefited from a conditional marketing authorisation for Duchenne muscular dystrophy, granted by the EMA for patients with a “nonsense” mutation of the DMD gene, aged 2 years and over, who are able to walk.
On 15 September 2023, the CHMP recommended that this marketing authorisation should not be renewed. This opinion is based on the analysis of all the data provided by PTC Therapeutics from phase II and III trials, other sub-group studies requested by the CHMP, and the STRIDE registry, which show in particular that :
- the efficacy of the drug was not confirmed in terms of walking distance in a sub-group of patients with a progressive reduction in walking capacity, after 18 months of treatment with translarna compared to placebo ;
- the results in the wider sense did not confirm the effects observed in the initial study which supported the marketing authorisation;
- although the real-life data (STRIDE registry) provided results on the preservation of walking, the benefits of translarna are not clear, in particular due to methodological problems of indirect comparison of treated and untreated populations.
While the European Commission’s final opinion on the continuation or discontinuation of the marketing authorisation is expected in the first quarter of 2024, PTC Therapeutics must submit a request for re-examination to the CHMP.
For the time being, patients receiving ataluren in France will be able to continue receiving it until at least this date.
EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna
EMA, press release, 15 September 2023
CHMP Delivers Opinion on Translarna™ for Conversion to Full Authorization
PTC Therapeutics, press release, 15 September 2023
