PTC Therapeutics announced on March 28, 2025, that the European Commission (EC) has adopted the negative opinion of the Committee for Medicinal Products for Human Use (CHMP). This decision results in the non-renewal of the conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy.
This action effectively removes Translarna's conditional marketing authorization in the European Economic Area. PTC Therapeutics expressed disappointment with the EC's decision following a prolonged review period.
However, 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 for the continued use of Translarna. PTC Therapeutics plans to work on a country-by-country basis to provide commercial drug where possible.
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