In Europe, the PTC Therapeutics drug Translarna, used to treat Duchenne muscular dystrophy, failed a confirmatory study but remains on the market for now. The European Commission declined to adopt a recommendation against renewing its marketing authorization. The drug received conditional approval in 2014 based on Phase 2 data and was up for renewal. The European Medicines Agency’s Committee initially recommended against renewal, but the commission returned the decision for reevaluation considering new evidence. Despite limited treatment options for Duchenne patients, Translarna sales increased in 2023. The commission’s decision is seen as unique and may signal a positive outcome for the drug’s renewal.
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European Commission Decision Is a Reprieve for PTC Therapeutics’ Rare Muscle Disease Drug
