Sarepta to Continue Shipping Gene Therapy Elevidys Despite FDA Request to Halt

Sarepta Therapeutics will continue shipping its gene therapy Elevidys for Duchenne muscular dystrophy despite an FDA request to halt shipments. The FDA requested the halt due to concerns over the therapy's effectiveness and safety. However, Sarepta announced it will continue shipping the treatment for ambulant patients.

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) has announced that it will continue shipping its gene therapy product Elevidys for the treatment of Duchenne muscular dystrophy (DMD) despite an FDA request to halt shipments. The decision comes amidst concerns over the therapy's safety and effectiveness.

The U.S. Food and Drug Administration (FDA) placed Sarepta's investigational gene therapy clinical trials for limb girdle muscular dystrophy on clinical hold following three deaths potentially related to these products. The FDA also revoked Sarepta's platform technology designation and requested the company to voluntarily stop all shipments of Elevidys [1].

Sarepta, however, has stated that it will continue shipping Elevidys to ambulant patients. The company asserts that its comprehensive scientific interpretation of the data shows no new or changed safety signals in the ambulant patient population. Sarepta has paused shipments for non-ambulant patients while it works with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure [2].

The FDA's concerns include the potential for acute liver failure in individuals treated with Elevidys or investigational gene therapy using the same AAVrh74 serotype. The FDA has also restricted the indication for Elevidys to use in ambulatory patients, pending verification and description of clinical benefit in a confirmatory trial [1].

Sarepta's commitment to patient safety and informed access to care remains a priority. The company is working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients, considering the compelling need for disease-modifying therapy [2].

References:
[1] https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold
[2] https://www.businesswire.com/news/home/20250718534546/en/Sarepta-Therapeutics-Provides-Statement-on-ELEVIDYS