FDA investigating risk of acute liver failure with Sarepta gene therapies

As of July 18, 2025, the Food and Drug Administration said it has received three reports of fatal acute liver failure following treatment of patients with Sarepta (SRPT) AAVrh74 gene therapies that appear to have been caused by the gene therapy products as a result of acute liver failure. The agency added, “FDA is investigating the risk of acute liver failure with serious outcomes, including those such as hospitalization and death, following gene therapies using Sarepta’s AAVrh74 gene therapy products, and the need for further regulatory action(s).”