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

 
• By Jessica Merrill and Mary Jo Laffler

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 approval is for a narrow indication in children 4-5 years old, and is based on a surrogate

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