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Gene Therapy
First gene therapy for Duchenne nets approval
by Shi En Kim
June 30, 2023
| A version of this story appeared in
Volume 101, Issue 21
The US Food and Drug Administration has conditionally approved Sarepta Therapeutics’ gene therapy Elevidys for Duchenne muscular dystrophy in children aged 4–5. Priced at $3.2 million, Elevidys delivers a gene that produces a shortened version of the dystrophin protein missing in people with the condition. In May, an FDA advisory panel narrowly voted to recommend approval, citing patients’ urgent unmet need for treatment options—despite Elevidys’s ambiguous benefit during its sole randomized and placebo controlled clinical trial.
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