Today the FDA announced a significant update to the labeling and indication of ELEVIDYS for the treatment of individuals living with Duchenne following reports of death based on acute liver failure (ALF) in non-ambulatory patients treated with the product. This comes after Sarepta shared in July 2025 that the agency requested and the company agreed to include a Boxed Warning (commonly referred to as a “black box” warning) for acute liver injury (ALI) and ALF be added to the ELEVIDYS label.
The FDA is also requiring Sarepta to conduct an additional post-marketing observational study to further assess the risk of serious liver injury. According to the agency, this study will enroll approximately 200 patients with Duchenne and focus on evaluating liver function for at least 12 months after administration of ELEVIDYS. We will be sure to update the community when details of this study are made available.
According to the FDA, the following labeling changes have been made after the agency’s review of available safety data:
- Limiting the indication to ambulatory patients with Duchenne who are 4 years of age and older with a confirmed mutation in the DMD gene;
- Removal of the indication for non-ambulatory patients with Duchenne;
- Addition of a Boxed Warning describing the risk of serious liver injury and ALF, including fatal outcomes;
- Addition of a Limitations of Use statement to guide clinical decision-making;
- Updates to the Warnings and Precautions, Dosage and Administration, Adverse Reactions, Use in Specific Populations, Clinical Studies, and Patient Counseling Information sections; and
- Inclusion of a new Medication Guide for patients and caregivers.
The revised labeling also includes the following specific safety information and monitoring recommendations:
- Liver monitoring: Weekly liver function tests are advised for at least three months after treatment. Patients should remain near an appropriate medical facility for at least two months post-infusion.
- Prompt medical attention: Patients should contact their health care provider immediately if they experience yellowing of the skin or eyes, if they miss or vomit corticosteroid doses, or if the patient experiences a change in mental status.
- Infection risk: Corticosteroid therapy may suppress immune function, increasing susceptibility to infections and serious complications including death.
- Cardiac monitoring: Weekly testing for cardiac injury (troponin-I) is advised for one month following treatment.
- Contraindications: ELEVIDYS should not be used in patients with deletions involving DMD exons 8 and/or 9.
- Limitations of Use: ELEVIDYS is not recommended in patients with pre-existing liver impairment, recent vaccinations, or recent/active infections.
This update serves as a reminder that while gene therapy breakthroughs bring promise and hope, serious questions remain about the safety and long-term outcomes of these therapies. If you have questions about your or your child’s care, please contact your neuromuscular team. We will share additional information with the community when available.
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