European Commission Dangles Conditional Approval Reprieve For PTC Therapeutics’ Translarna

In a very rare move, the European Commission has rejected advice from the European Medicines Agency not to renew the conditional marketing authorization (CMA) for PTC Therapeutics’ rare disease treatment Translarna (ataluren), the first ever treatment approved for Duchenne muscular dystrophy.

The commission has asked the EMA to review its scientific assessment of the drug following scientific questions from EU member state experts and in light of both new real-world evidence and a recent court ruling relating to the impartiality of