Updated: FDA approves Sarepta’s Duchenne muscular dystrophy gene therapy via accelerated pathway
The FDA granted accelerated approval to Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy for boys aged 4 and 5 in a decision that allows access to the first one-time treatment for the debilitating muscle disease.
The gene therapy, known as SRP-9001 or delandistrogene moxeparvovec, will be marketed as Elevidys, Sarepta announced Thursday afternoon. Elevidys will cost $3.2 million per patient, making it the second most expensive medicine in the US after CSL Behring and uniQure’s hemophilia B gene therapy Hemgenix, which costs $3.5 million per patient.
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