Sarepta基因疗法Elevidys在两名患者死亡后收到FDA盒装警告

2025-11-15 05:24

The US FDA has ordered a Boxed Warning be added to the labeling of Sarepta Therapeutics' (SRPT) Elevidys (delandistrogene moxeparvovec) about the risk of fatal acute liver failure for the gene therapy used for Duchenne muscular dystrophy.
The labeling is also being updated for use only in ambulatory patients ages four and higher.
Sarepta reported the deaths of two teenage boys who were non-ambulatory who used the therapy, first in March and then in June. In July, the company said a third patient who was taking part in an early-stage trial for a new treatment for limb-girdle muscular dystrophy died from acute liver failure.
"In both fatal cases, patients developed markedly elevated liver enzymes and required hospitalization within two months of Elevidys infusion," the FDA said in a press release.
The agency also removed the indication for Elevidys in non-ambulatory patients.
Sarepta shares have suffered mightily in 2025 as a result of the deaths, and are down ~85% year to date.

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