Sarepta reports second case of liver failure death after its gene therapy treatment
June 15 (Reuters) - Sarepta Therapeutics (SRPT.O), opens new tab on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy for a rare form of muscular dystrophy.
The patient was undergoing treatment with Sarepta's therapy called Elevidys, which is the only gene therapy approved by the U.S. Food and Drug Administration for Duchenne muscular dystrophy patients aged four and above.
The first instance of death was reported in March this year, a 16-year-old boy who died from acute liver failure months after receiving Elevidys.
Liver damage is a known risk with Elevidys and other gene therapies that use adeno-associated viral vectors to infuse modified genes.
Sarepta said it is taking steps to mitigate the risk of acute liver failure in patients by working to convene an independent group of experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for Elevidys. It has also temporarily suspending shipments of the therapy and informed the FDA and global health authorities about the incident.
Roche (ROG.S), opens new tab, which partners with Sarepta for commercialization of the gene therapy outside the United States, said in a separate statement on Sunday that it has paused the dosing of Elevidys in patients following the two cases of fatal acute liver failure.
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