Letter to the community added November 17, 2025
The FDA has approved the new labeling information Sarepta provided for Elevidys, Sarepta’s gene therapy for Duchenne. This update comes after reports of fatal acute liver failure in non-ambulatory patients, and a review of all of the available safety data.
The updates include:
- A Boxed Warning for the risk of serious liver injury and acute liver failure, including fatal outcomes
- Limiting the indication to ambulatory individuals (aged 4 years and older) only. The non-ambulatory indication has been removed.
- Various additions to help guide prescriber’s clinical decision making, including modified pre- and post- infusion corticosteroid regimen, and enhanced monitoring recommendations for 3 months post-infusion
- A new Warnings & Precautions regarding increased susceptibility to serious infections
- A new Medication guide for patients and caregivers
The FDA has also asked Sarepta to conduct a postmarketing observational study in 200 individuals and follow them for at least a year after treatment, with periodic liver assessments to further assess the risks.
For non-ambulatory individuals, Sarepta has indicated they plan to work with the FDA to commence a study using an enhanced immunosuppressive regimen using sirolimus, in conjunction with Elevidys dosing.
Link to Sarepta’s press release:
Link to FDA News release:
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