Research & Development
PTC's Translarna faces setback in Europe
28 June 2024 -

US-based biopharmaceutical company PTC Therapeutics Inc (NASDAQ:PTCT) announced on Friday a negative opinion from the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) on the renewal of the conditional marketing authorisation of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

This follows the return of the previously issued negative opinion by the European Commission for re-review.

PTC intends to request re-examination of the opinion.

The marketing authorisation remains valid until the end of 2024, even if the negative opinion is maintained and ratified.

PTC argues that real-world evidence supports Translarna's use and said it will fight to keep it available for DMD patients.