The FDA said Nov. 14 it will place its most prominent safety warning on Elevidys, a gene therapy for Duchenne muscular dystrophy, after two fatal cases of acute liver injury.
Sarepta Therapeutics paused shipments of the therapy to nonambulatory patients in June — two years after the drug’s approval. In July, the Cambridge, Mass.-based biotech company stopped all deliveries as it discussed possible safety labeling changes with the FDA.
Officials have reported three deaths following treatment with Elevidys, two of which the FDA attributed to the gene therapy. In both cases, “patients developed markedly elevated liver enzymes and required hospitalization within two months of Elevidys infusion,” according to the agency. A nonfatal case of acute liver injury was also reported.
In a Nov. 14 statement, the FDA said it is working to approve new safety labeling submitted by Sarepta, including a boxed warning and a limited indication. The amended indication only includes ambulatory patients.
Elevidys is the only gene therapy approved for Duchenne muscular dystrophy. More than 900 individuals have received Elevidys through clinical trials and commercial use, according to Sarepta.
In addition to the safety label and indicated use parameters, the FDA will require Sarepta to conduct a 200-person, 12-month study to assess the risk of serious liver injury. Following treatment, healthcare providers are advised to perform weekly liver function tests for at least three months and weekly testing for cardiac injury for one month.
