Sarepta Pushes Back on FDA as Agency Reacts to Patient Deaths

Rare Daily Staff

After reports of the deaths of two non-ambulatory people treated with Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, Elevidys, and a third person treated with one of the company’s experimental limb girdle muscular dystrophy gene therapies, the U.S. Food and Drug Administration asked the company to voluntarily pull Elevidys from the market and placed a clinical hold on all of its limb-girdle muscular dystrophy programs.

Despite the FDA request, Sarepta said it will continue to ship Elevidys for ambulatory people with Duchenne.

The three deaths appear to have been a result of acute liver failure in individuals treated with gene therapies using the same AAVrh74 serotype.

“Today, we’ve shown that this FDA takes swift action when patient safety is at risk,” said FDA Commissioner Marty Makary. “We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges.”

In a statement it released, Sarepta said it first learned of the FDA request through media reports at the same time as the public and its patient communities.

The company noted it made the “conservative decision” to pause shipments of Elevidys for non-ambulatory patients while it works with the agency to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure.

“Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulatory patient population, we will continue to ship Elevidys to the ambulatory population,” the company said.

Separately, Sarepta said that the U.S. Food and Drug Administration placed a clinical hold on the company’s experimental gene therapy clinical trials for limb-girdle muscular dystrophy, including those related to its product candidates for the condition, according to a filing with the U.S. Securities and Exchange Commission.

Sarepta previously announced on July 16, 2025, that it had paused each of the limb-girdle muscular dystrophy programs as part of a strategic restructuring process, with the exception of SRP-9003, which completed enrollment and dosing in a phase 3 study.

The company said it will seek a discussion with the FDA about the potential pathway to seek accelerated approval from the FDA for SRP-9003 after the clinical hold is lifted.

In the same filing, Sarepta reported that the FDA has revoked the platform technology designation for the company’s AAVrh74 platform technology, which was granted in June.

Photo: FDA Commissioner Marty Makary