Washington, D.C. (WDNews) – The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a gene therapy used to treat Duchenne muscular dystrophy.
The child died on June 7, 2025, after receiving the therapy developed by Sarepta Therapeutics. In response to the incident, the FDA has requested and received a voluntary suspension of Elevidys distribution while safety concerns are reviewed.
Elevidys is a one-time intravenous treatment that uses an adeno-associated virus (AAV) vector to deliver a modified gene intended to slow the progression of Duchenne, a rare genetic disorder that causes progressive muscle weakening and typically affects boys.
The FDA has not released further details pending the outcome of the investigation. Sarepta’s AAVrh74 gene therapy platform has previously been approved for limited use under strict conditions.
