With apparently the first death of a patient dosed with its gene therapy Elevidys (delandistrogene moxeparvovec), Sarepta incurred a significant reduction in its stock price on 18 March, as well as questions about how new safety concerns around its $3.2m-priced product, the first gene therapy for Duchenne muscular dystrophy (DMD), will affect usage going forward.
Key Takeaways
- Sarepta said a DMD patient given Elevidys died of acute liver injury, but noted that more than 800 patients have been dosed with the gene therapy with no other reported deaths.
- The company’s stock price declined more than 27% on the news, two months after Sarepta reported that Elevidys brought in $1.79bn in sales during 2024
Before the US stock market opened on 18 March, Sarepta reported that a young male DMD patient treated with Elevidys died from acute liver failure. It later was revealed that the patient was 16 years old and died in December
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