EC calls for CHMP to re-evaluate Translarna opinion

The European Commission (EC) has decided not to adopt the European Medicines Agency’ human medicines committee CHMP's negative opinion of January 24, 2024 on the annual renewal of the conditional marketing authorization of Translarna (ataluren) and has returned the opinion to the CHMP for re-evaluation.

The Duchenne muscular dystrophy medicine is marketed by USA-based rare disease drug developer PTC Therapeutics (Nasdaq: PTCT), whose shares closed up more than 21% at $40.12 on the news yesterday.

As a result, Translarna remains on the market and available for patients in Europe consistent with its current marketing authorization. The EC has asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion.