(Reuters) -Sarepta Therapeutics said on Friday it had temporarily halted three trials testing its gene therapy Elevidys, following the death of a patient last month upon receiving the treatment.
The company reported on March 18 that a 16-year-old boy died from acute liver failure months after receiving the U.S.-approved gene therapy for a rare muscular dystrophy.
Sarepta said an independent monitoring committee met on April 3 to review the safety event and concluded that “the overall benefit-risk profile remains favorable to continue dosing in the paused clinical trials without changes to the study protocols”.
At the request of European Union regulators, Sarepta and partner Roche will submit this information within a week to lift the halt.
Sarepta said it would continue monitoring and data collection for already-enrolled participants. The company does not expect a material impact on the timeline for these studies.
Elevidys is the only gene therapy approved by the U.S. Food and Drug Administration for Duchenne muscular dystrophy patients aged four and above.
Liver damage is a known risk with Elevidys and other gene therapies that use adeno-associated viral vectors to infuse modified genes.
(Reporting by Christy Santhosh in Bengaluru; Editing by Shilpi Majumdar)
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