In May 2024, PTC Therapeutics had been announced that the European Commission (EC) has decided not to adopt the CHMP’s negative opinion on the annual renewal of Translarna’s conditional marketing authorization (ataluren) and has returned the opinion to the CHMP for re-evaluation.
European Commission Declines To Renew Marketing Authorization Of Translarna Duchenne Muscular Dystrophy
PTC Therapeutics, announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy. [What is Ataluren?]
While this action effectively removes the drug’s conditional marketing authorization in the European Economic Area, the EC indicated that individual countries within the European Union can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued use of Translarna.
PTC Therapeutics said Duchenne Drug Ataluren is No Longer Authorized in EU
“We are of course disappointed that after this prolonged period of review the European Commission has decided to adopt the CHMP negative opinion on Translarna,” said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. “The EC’s indication that European Union member states have a mechanism to maintain treatment speaks to the safety, benefit and lack of alternative therapies for boys and young men with nonsense mutation Duchenne muscular dystrophy. We look forward to working on a country-by-country basis to provide commercial drug where possible.”
Read More: Call from Families to European Medicines Agency (EMA): Re-examine Ataluren (Translarna)
