On 28 March 2025, the European Commission announced the withdrawal of the European marketing authorisation for Translarna® in Duchenne muscular dystrophy. This concerned DMD patients aged 2 and over who were still walking and had a nonsense genetic anomaly in the DMD gene.
In France, the decision was taken on 17 June 2025 by the Ministers of Health and Social Security, following advice from the ANSM, to end the derogatory reimbursement of Translarna® by the Social Security schemes. PTC Therapeutics France has sent a letter to healthcare professionals informing them of this decision.
Translarna (ataluren) : arrêt de mise à disposition suite au non‐renouvellement de l’AMM conditionnelle européenne (in French), Agence Nationale de sécurité du médicament et de produits de santé (ANSM), 17 June 2025
PTC Therapeutics Provides Regulatory Update on Translarna™ (ataluren) in Europe, PTC Therapeutics, press release, 28 March 2025
Commission implementing decisin, 28 March 2025
