The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had decided twice against renewing the marketing authorisation for Translarna (ataluren) for Duchenne muscular dystrophy (DMD). In May 2024, the European Commission annulled the procedure on the grounds of procedural irregularities. It asked the EMA to re-analyse the available evidence, supplemented by more recent publications.
Following this new examination, the CHMP once again concluded that Translarna was not effective and that the benefit/risk ratio was not favourable to renewal of the conditional marketing authorisation. The company has again appealed against this decision, which means that a final decision on whether or not to continue the marketing authorisation will be taken at the end of 2024.
EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna – EMA, 28 June 2024 press release
PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation – PTC Therapeutics, 20 May 2024 press release
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
