On July 18, 2025, the U.S. Food and Drug Administration (FDA) took decisive action against Sarepta Therapeutics following the deaths of three individuals who had received the company’s gene therapy products. As reported by Drugs.com, the agency placed Sarepta’s clinical trials for limb girdle muscular dystrophy (LGMD) on hold, citing new safety concerns and the potential for significant risk of illness or injury. Additionally, the FDA revoked Sarepta’s platform technology designation for its AAVrh74-based therapies. Despite the FDA’s request to stop shipments of Elevidys—Sarepta’s approved gene therapy for Duchenne muscular dystrophy (DMD)—the company refused.
FDA Commissioner Marty Makary emphasized the agency’s commitment to patient safety, stating, “We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges.” The three patient fatalities were all due to acute liver failure. Each had been treated with Elevidys or similar investigational gene therapies utilizing the AAVrh74 viral vector, a platform technology designed to deliver therapeutic genes to muscle cells.
Elevidys, which received FDA approval for ambulatory DMD patients in June 2024 and for non-ambulatory patients under accelerated approval in June 2023, works by providing the genetic code for Elevidys micro-dystrophin—a shortened but functional version of the dystrophin protein that is absent or abnormal in DMD patients. The therapy is administered as a single intravenous dose, with the goal of preserving muscle function in a disease that otherwise leads to progressive muscle weakness and loss.
Duchenne muscular dystrophy is a rare, inherited disorder marked by rapid muscle degeneration due to mutations in the DMD gene. The absence of dystrophin makes muscle cells fragile and prone to damage. Elevidys was seen as a breakthrough for these patients, offering hope for improved quality of life.
However, the emergence of severe, potentially fatal liver complications has shifted the risk-benefit balance. According to Dr. Vinay Prasad, Director of the FDA’s Center for Biologics Evaluation and Research, “The FDA will not allow products whose harms are greater than benefits. We will halt any clinical trial if participants would be exposed to unreasonable and significant risk.”
The FDA’s revocation of Sarepta’s platform technology designation was based on insufficient evidence that the AAVrh74 vector could be safely used across multiple therapies. As a further precaution, the agency restricted Elevidys’s indication to ambulatory DMD patients only, pending further investigation.
The agency is actively investigating the risk of acute liver failure with gene therapies using Sarepta’s platform and considering further regulatory actions. The FDA reaffirmed its commitment to protecting patients and will take additional steps as necessary to ensure safety.
