Death of 8-year-old not tied to Sarepta gene therapy: FDA – Becker’s Hospital Review | Healthcare News

The FDA has reported that the recent death of an 8-year-old boy was unrelated to Sarepta Therapeutics’ gene therapy Elevidys and recommended lifting the voluntary hold on the treatment, which is used for Duchenne muscular dystrophy. 

The agency paused use of the gene therapy for the patient group after the boy died June 7, launching a safety investigation and prompting Sarepta to temporarily halt distribution. Sarepta had halted shipments of Elevidys July 21 as part of an agreement with the FDA to update the safety labeling and complete a regulatory review process. 

The FDA’s latest update clears Elevidys for use in ambulatory Duchenne muscular dystrophy patients, but a voluntary hold remains in place for nonambulatory patients following two other deaths earlier this year linked to the therapy, according to a July 28 news release from the agency. 

“Last week, at the suggestion of FDA, Sarepta made the difficult decision to pause shipments of Elevidys to provide the FDA with an opportunity to complete a review of available safety information. We are very pleased that FDA chose to rapidly and comprehensively complete that review and to recommend that we remove our voluntary pause and resume shipment of ELEVIDYS for ambulatory patients. The FDA’s swift review evinces a commitment to the Duchenne population, a commitment shared by Sarepta,” Doug Ingram, chief executive officer at Sarepta, said in a news release July 28.