FDA Adds Boxed Warning to Duchenne Muscular Dystrophy Gene Therapy Elevidys Following Liver Failure Reports
The U.S. Food and Drug Management on Thursday, November 14, 2025, announced significant revisions to the labeling of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for duchenne muscular dystrophy (DMD), including the addition of a Boxed Warning – the agency’s most prominent safety alert. These actions come after reports of fatal acute liver failure in patients receiving the treatment.
The FDA’s decision limits the approved use of Elevidys to ambulatory patients aged four years and older with a confirmed genetic mutation causing DMD.The agency previously approved Elevidys as a treatment for a broader range of DMD patients,but has now removed that indication for those who are non-ambulatory.
Elevidys and the Emergence of Safety Concerns
Elevidys is an adeno-associated virus (AAV) vector-based gene therapy designed to address the underlying genetic defect in certain DMD patients. In june 2025, the FDA’s Center for Biologics Evaluation and Research (CBER) issued a safety interaction following two reported cases of fatal acute liver failure.
Key Updates to Elevidys Labeling and Usage
- Boxed Warning: A prominent warning detailing the risk of serious liver injury and acute liver failure, including potentially fatal outcomes.
- Restricted Indication: Elevidys is now only approved for ambulatory DMD patients four years of age and older with a confirmed mutation in the DMD gene.
- Removal of Non-Ambulatory Indication: The previous indication for non-ambulatory patients has been removed.
- Limitations of Use Statement: A statement to guide clinicians in appropriate patient selection.
- Updated Sections: Revisions to Warnings and Precautions, Dosage and Administration, Adverse Reactions, Use in Specific Populations, Clinical Studies, and Patient Counseling Information.
- Medication Guide: A new guide for patients and caregivers outlining key safety information.
The FDA is emphasizing the importance of close monitoring for patients receiving Elevidys. Key safety recommendations include:
- Liver Monitoring: Weekly liver function tests are advised for at least three months post-treatment, with patients remaining near a medical facility for at least two months.
- Prompt Medical Attention: Patients should instantly contact their healthcare provider if they experience yellowing of the skin or eyes, miss or vomit corticosteroid doses, or experience a change in mental status.
- Infection Risk: Corticosteroid therapy, often used in conjunction with Elevidys, can suppress immune function, increasing susceptibility to infections.
- Cardiac Monitoring: Weekly testing for cardiac injury (troponin-I) is recommended 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: The therapy is not recommended for patients with preexisting liver impairment, recent vaccinations, or recent/active infections.
Postmarketing Surveillance and Adverse Event Reporting
The FDA is requiring Sarepta Therapeutics to conduct a postmarketing observational study, enrolling approximately 200 DMD patients, to further assess the risk of serious liver injury. Participants will be followed for at least 12 months with periodic liver function assessments.
Healthcare professionals and patients are encouraged to report any adverse events, particularly cases of liver injury, to the FDA’s MedWatch program. Reports can also be made directly to Sarepta Therapeutics at 1-888-727-3782.
The FDA affirmed its commitment to ongoing evaluation of gene therapies, stating it will provide updates as new information becomes available. This case underscores the complex balance between innovation and patient safety in the rapidly evolving field of gene therapy.
