A Momentous Day For DMD: Sarepta’s Elevidys Is First Gene Therapy Approved

 
• By Jessica Merrill and Mary Jo Laffler
  
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The US FDA granted an accelerated approval to Sarepta’s Elevidys, the first gene therapy to be approved for some children with Duchenne muscular dystrophy.

IV solution in a child's patients hand

Sarepta Therapeutics, Inc. is confident its newly approved gene therapy for Duchenne muscular dystrophy (DMD), Elevidys (delandistrogene moxeparvovec-rokl), will be a commercial success despite initial age restrictions and carrying a price tag of $3.2m. The company has worked at being transparent around pricing and believes “the work that we've done to support the pricing of this therapy is potentially a blueprint for others,” CEO Doug Ingram said during a 22 June analyst call about the approval.

The US Food and Drug Administration granted an accelerated approval to Sarepta’s gene therapy earlier in the day, though the...

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