WASHINGTON, D.C. — The U.S. Food and Drug Administration on Friday placed Sarepta Therapeutics’ gene therapy trials for limb girdle muscular dystrophy on clinical hold following the deaths of three patients and the emergence of serious safety concerns. The agency also revoked Sarepta’s platform technology designation for its AAVrh74 vector system, citing insufficient evidence of safety across multiple applications.
The decision comes amid growing unease surrounding Elevidys, an adeno-associated virus (AAV) vector-based gene therapy developed by Sarepta for the treatment of Duchenne muscular dystrophy (DMD). All three fatalities are believed to be linked to acute liver failure in patients who received either Elevidys or an investigational therapy utilizing the same viral delivery platform.
FDA Commissioner Dr. Marty Makary underscored the agency’s decisive response: “Today, we’ve shown that this FDA takes swift action when patient safety is at risk. 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 agency confirmed that one of the three deaths occurred during a trial for limb girdle muscular dystrophy, an investigational use of the same AAVrh74 vector platform. In response, the FDA asked Sarepta to voluntarily cease all shipments of Elevidys—a request the company declined.
Dr. Vinay Prasad, Director of the FDA’s Center for Biologics Evaluation and Research, reinforced the agency’s position: “Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits. The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury.”
Elevidys, which delivers a micro-dystrophin gene via a single intravenous dose, received traditional FDA approval for ambulatory DMD patients aged 4 and older in June 2024. An earlier accelerated approval for non-ambulatory patients was granted in 2023. In light of the new safety data, the agency has moved to limit the product’s use strictly to ambulatory patients, pending further investigation.
The FDA also announced the revocation of Sarepta’s AAVrh74 platform designation. The decision reflects the agency’s determination that the platform no longer demonstrates sufficient safety to justify broader application across multiple gene therapies.
Duchenne muscular dystrophy is a progressive and debilitating genetic disorder caused by the absence or malfunction of dystrophin, a protein critical to muscle cell integrity. Elevidys aims to address this deficit with a shortened form of the protein, though questions remain about its long-term efficacy and safety.
The FDA’s investigation into risks associated with the AAVrh74 platform is ongoing. Additional regulatory actions may follow as the agency continues to assess the threat of acute liver failure and other adverse outcomes linked to Sarepta’s gene therapies.
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