Action Duchenne Expresses Deep Concern Over CHMP’s Negative Opinion on Translarna™ for Duchenne Muscular Dystrophy
Action Duchenne expresses profound concern following the recent decision by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) regarding Translarna™ (Ataluren).
PTC Therapeutics announced that CHMP has given a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). This decision also applies to the renewal of the existing conditional authorization.
PTC plans to submit a request for re-examination per EMA guidelines. Translarna will remain on the market and available to patients with DMD until the re-examination process is completed.
Action Duchenne firmly stands by the importance of Translarna™ as a potential therapeutic option for Duchenne individuals with nonsense mutation. The negative opinion from CHMP has left our community deeply concerned about the future availability of this treatment option.
In these challenging times, Action Duchenne reiterates its commitment to supporting families affected by Duchenne muscular dystrophy. We will closely monitor developments related to Translarna™ and advocate tirelessly for its approval, ensuring that individuals living with Duchenne continue to have access to treatments that can improve their quality of life.
Read the Company Press Release here
Read the EMA Recommendation here
Do you want to know more?
The Annual Action Duchenne International Conference 2023 will be held on the 10th and 11th November and is an amazing opportunity to meet with those involved in every aspect of Duchenne, from families, clinicians, researchers and pharmaceutical companies. Tickets are FREE for Duchenne families as well as everyone living with Duchenne AND there is a £100 grant available for Duchenne families towards travel and accommodation. Book your place now.
