Conditional EMA approval given for Translarna for Duchenne Muscular Dystrophy - PTC Therapeutics
The European Medicines Agency has granted under a special exemption known as a conditional marketing authorization, approval for Translarna (ataluren) from PTC Therapeutics for Duchenne Muscular Dystrophy. A conditional approval is intended to allow for medicines that may combat life-threatening illnesses when there are no other drug options available.
EMA which had denied approval in January 2014, said it was issuing the new decision after a re-examination of the evidence and new data from PTC Therapeutics. There are currently no other treatments for the disease, which primarily effects boys and is the result of a mutational defect in genes that prematurely ends production of dystrophin.
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