The US Food and Drug Administration (FDA) has placed gene therapy clinical trials on hold following three deaths potentially related to Sarepta Therapeutics’ investigational product Elevidys, used to treat limb girdle muscular dystrophy. The FDA also revoked the platform technology designation for Sarepta’s AAVrh74 Platform Technology due to new safety concerns.
The agency requested Sarepta to voluntarily stop all shipments of Elevidys but the company refused. FDA Commissioner Marty Makary stated that patient safety is the top priority and that the agency will take immediate action when a serious safety signal emerges.
Elevidys is an adeno-associated virus vector-based gene therapy designed to deliver a specific gene into the body to produce a shortened version of the dystrophin protein. However, this protein has been linked to acute liver failure in individuals treated with Elevidys or similar gene therapies.
The FDA will restrict the use of Elevidys to ambulatory patients and further investigate the safety of the product in these patients. The agency is committed to protecting patients and taking additional steps if necessary.
This decision comes after a series of deaths associated with gene therapy products using Sarepta’s AAVrh74 Platform Technology. The FDA will continue to monitor the risk of acute liver failure and take regulatory actions as needed to ensure patient safety.
Source: https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold