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.
European Commission Dangles Conditional Approval Reprieve For PTC Therapeutics’ Translarna
Scientific questions from member state experts and a recent court ruling on impartiality in the drug approval process prompted the European Commission to query the EMA’s negative opinion on PTC’s Translarna.
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