Sarepta Therapeutics Updates ELEVIDYS Label and Enhances Safety Measures

Sarepta Therapeutics has updated the label for its gene therapy ELEVIDYS to include a black box warning for acute liver injury and acute liver failure, as requested by the FDA. The company has also convened an expert committee to explore additional immunosuppression regimens for non-ambulant patients and will submit findings to the FDA. Sarepta plans to discuss a proposal for a new cohort of the ENDEAVOR study and assess real-world data generation opportunities for ambulant patients.

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) has announced significant updates to its gene therapy ELEVIDYS (delandistrogene moxeparvovec) and a strategic restructuring plan to bolster its financial foundation. The company has updated the ELEVIDYS label to include a black box warning for acute liver injury (ALI) and acute liver failure (ALF), as requested by the U.S. Food and Drug Administration (FDA) [1].

In response to these changes, Sarepta has convened an Expert Committee comprising neuromuscular specialists, hepatologists, hematologists, and immunologists to review cases of ALF and explore additional immunosuppression regimens. The committee has aligned on an enhanced immunosuppressive regimen with sirolimus for ELEVIDYS in non-ambulant patients. Sarepta will submit the findings and proposed protocol to the FDA and discuss a proposal to gather data on the regimen in a new cohort (Cohort 8) of the ENDEAVOR study (Study SRP-9001-103) [1].

Additionally, Sarepta is assessing real-world data generation opportunities for ambulant patients through investigator-initiated trials. The company is also focusing its pipeline on high-impact programs, primarily leveraging its potentially best-in-class siRNA platform. This strategic pivot emphasizes chronically administered therapies for neurodegenerative and pulmonary diseases [1].

As part of its restructuring plan, Sarepta has initiated immediate changes to reduce operating expenses and align its cost structure with strategic priorities. These measures are projected to deliver approximately $400 million in annual cost reductions, significantly lowering the Company’s average annual non-GAAP R&D and SG&A expenses to between $800 million and $900M starting in 2026 [1].

Sarepta will host an investor call on Wednesday, July 16, 2025, at 4:30 p.m. Eastern time, to discuss these updates. The event will be webcast live under the investor relations section of Sarepta's website at: https://investorrelations.sarepta.com/events-presentations [1].

References:
[1] https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-strategic-restructuring-and