Sarepta Therapeutics Inc. (NASDAQ: SRPT) received an informal request from the U.S. Food and Drug Administration to voluntarily halt shipments of ELEVIDYS, its gene therapy for Duchenne muscular dystrophy, according to a company statement.

The FDA request came after 2:30 p.m. ET, with Sarepta stating it first learned of the potential request through media reports earlier that day. The company had already paused shipments to non-ambulant patients while working with the FDA to update labeling and evaluate enhanced immunosuppression protocols to mitigate acute liver failure risks.

Sarepta said it will continue shipping ELEVIDYS to ambulant patients, citing no new or changed safety signals in that population based on its analysis of the data.

The company clarified that a recent death of a 51-year-old non-ambulant patient with Limb-Girdle Muscular Dystrophy occurred during a Phase 1 clinical trial for SRP-9004, a different investigational gene therapy. The patient was not treated with ELEVIDYS, and dosing for the SRP-9004 trial had concluded at the time of death.

Sarepta reported the acute liver failure event to the FDA on June 20, 2025, and notified the agency of the death on July 3, 2025, according to the statement.

ELEVIDYS is approved for treating Duchenne muscular dystrophy in patients at least 4 years old with confirmed DMD gene mutations. The therapy received accelerated approval for non-ambulant patients based on expression of micro-dystrophin in skeletal muscle, with continued approval contingent on confirmatory trials demonstrating clinical benefit.

The gene therapy carries warnings for infusion-related reactions, acute serious liver injury, immune-mediated myositis, myocarditis, and issues related to preexisting immunity against the AAV vector used in the treatment.