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European Medicine Agency’s Committee for Medicinal Products for Human Use Issues Negative Opinion on Translarna Following European Commission Request for Review

1 July 2024

On Friday 28 June 2024 PTC Therapeutics announced that the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion on the renewal of the conditional marketing authorisation of Translarna for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation. This opinion follows the return of the previously issued negative opinion by the European Commission (EC) for re-review, which we reported in May. 

In its announcement, PTC confirmed plans to request re-examination of the opinion and stated that the marketing authorisation for Translarna remains in effect, pending the outcome of this process and ratification of the final decision by the EC. Based on its understanding of the timeline of these procedures, PTC expects Translarna to remain on the market in Europe through to the end of 2024 even if the negative opinion is maintained and ratified. 

For more information, please refer to the PTC Therapeutics press release, available at CHMP Issues Negative Opinion on Translarna™ Following European Commission Request for Review | PTC Therapeutics, Inc. (

Following Brexit, the EMA does not have authority over UK marketing authorisation. This is the responsibility of the Medicines and Healthcare products Regulatory Agency (MHRA). The MHRA has begun its own review of Translarna, which we are engaging in to represent your views and experiences. Translarna remains available in the UK and we will keep the you informed as further developments take place or announcements are made. 

In February 2023, NICE recommended Translarna as a treatment for children aged two and over with Duchenne muscular dystrophy, making it available on the NHS in England, Wales and Northern Ireland. We acted as a patient expert in the appraisal process, presenting the views of the Duchenne community after extensive  consultation and engagement. Read the full NICE guidance. 

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