FDA Limits Gene Therapy Treatment Following Deaths of 2 Children

The drug manufacturer has been instructed to include and highlight the adverse health impacts on the label.

The Food and Drug Administration building in White Oak, Md., seen on June 5, 2023.Madalina Vasiliu/The Epoch Times

The Food and Drug Administration approved an updated safety warning for a gene therapy treatment that restricts its use following reports of two deaths among pediatric users, the agency said in a statement on Nov. 14.

The approval was granted for Elevidys, a one-time gene therapy treatment for Duchenne muscular dystrophy (DMD) by pharmaceutical company Sarepta Therapeutics Inc. DMD renders muscle cells vulnerable to injury, leading to progressive muscle degeneration.