FDA Reviewing Elevidys Black Box Warning About Acute Liver Injury Risk

Sarepta Therapeutics stopped shipping the Duchenne muscular dystrophy treatment in July 2025, following an FDA request.

A black box warning for the gene therapy Elevidys is now under federal review after the treatment was linked to at least three patient deaths.

Elevidys (delandistrogene moxeparvovec-rokl) was developed by Sarepta Therapeutics as a one-time gene therapy for children age 4 and older with Duchenne muscular dystrophy (DMD), a rare genetic condition caused by the body’s inability to produce dystrophin, a protein essential for healthy muscle function. 

The therapy became the first gene treatment authorized for DMD when the U.S. Food and Drug Administration (FDA) granted accelerated approval in June 2023, a decision that drew controversy after senior FDA leadership overruled internal reviewers who questioned whether the evidence was sufficient. The agency later expanded the indication in 2024, making most U.S. patients with DMD eligible to receive it.

However, concerns escalated in June 2025 when federal regulators issued a safety alert after two non-ambulatory pediatric patients developed acute liver failure shortly after infusion. At the FDA’s request, Sarepta halted distribution of Elevidys for non-ambulatory use, and mounting reports of patient deaths prompted a broader regulatory review that culminated in the announcement of a black box warning in July.

In a press release issued on November 14, the FDA now says that it has begun the formal approval process for the boxed warning submitted by Sarepta.

As part of the warning, the new labeling will include detailed safety instructions, such as:

• Liver monitoring: Patients should undergo weekly liver function testing for at least three months after receiving Elevidys, and are advised to stay within reach of a medical facility for the first two months following infusion.
• Seek immediate care: Caregivers should contact a health care provider right away if the patient develops jaundice, misses or vomits a corticosteroid dose, or shows any sudden changes in behavior or alertness.
• Infection concerns: Corticosteroids can weaken the immune system, so patients may face a higher risk of infections and potentially life-threatening complications.
• Heart monitoring: Weekly troponin-I tests are recommended for the first month after treatment to watch for signs of cardiac injury.
• Contraindications: Elevidys should not be given to patients with deletions that affect DMD gene exons 8 or 9.
• Use limitations: The therapy is not advised for individuals with existing liver problems, those who recently received vaccinations, or patients with current or recent infections.

In addition to the Elevidys black box warning, federal regulators are also recommending the following changes in the product’s use guidelines:

• Restricting treatment to ambulatory patients with DMD who are at least 4 years old and have a confirmed DMD gene mutation
• Removing approval for use in non-ambulatory DMD patients
• Adding a Limitations of Use section to help guide prescribing decisions
• Updating multiple sections of the label, including Warnings and Precautions, Dosage and Administration, Adverse Reactions, Use in Specific Populations, Clinical Studies and Patient Counseling Information
• Requiring a new Medication Guide to better inform patients and caregivers about risks and proper use

The FDA has also ordered Sarepta to carry out a postmarketing observational study to better evaluate the drug’s potential to cause severe liver damage. The study is expected to include about 200 individuals with DMD, who will be monitored for at least a year after receiving Elevidys, including regular liver function testing throughout the follow-up period.

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