Sarepta adds boxed warning to Elevidys about risk of liver disease

The FDA and Sarepta Therapeutics have updated the prescribing information of Elevidys (delandistrogene moxeparvovec-rokl), an approved gene therapy to treat patients with Duchenne muscular dystrophy. A black box warning has been added to warn about the risk of acute serious liver injury (ALI) and acute liver failure (ALF), and the indication for non-patients has been removed. Elevidys is now indicated only to treat ambulatory patients four years of age and older.

Related: FDA Wants Elevidys Off the Market. Sarepta Said No.

In March of this year, Sarepta had announced the death of a teenager with Duchenne muscular dystrophy following treatment with Elevidys. This triggered an investigation by the FDA, with regulators demanding that the company stop shipment of the gene therapy. In June 2025, the FDA issued a CBER Safety Communication following two reports of fatal acute liver failure in non-ambulatory pediatric males with DMD after receiving Elevidy, and the FDA also placed on hold a clinical trial of Elevidys to treat limb-girdle muscular dystrophy. Sarepta did agree to a temporary halt in shipments to maintain a working relationship with regulators.

Regulatory had been concerned about Sarepta’s AAVrh74 platform technology used in Elevidys and another investigational gene therapy in a phase 1 trial where a patient also died. In an investor call in July, Ingram said the company had decided not to proceed with further development of SRP-9004. That decision, however, was not made based on the safety of the therapy or issues related to acute liver failure. The company is prioritizing instead its siRNA platform.

Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) technology to deliver a gene designed to produce a shortened form of the dystrophin protein, known as Elevidys micro-dystrophin, directly to skeletal muscle. Elevidys is priced at $3.2 million per dose. Sarepta had $1.7 billion in net product revenue in 2024, almost half of which — $821 million — came from Elevidys.

The new label includes several changes, including:

• The addition of a Boxed Warning describing the risk of serious liver injury and acute liver failure, including fatal outcomes;
• Limiting the indication to ambulatory patients with DMD 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 DMD;
• 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 specific information about monitoring patients who receive Elevidys and includes weekly liver function tests for at least three months, as well as weekly monitoring for cardiac injury for one month.

Additionally, the FDA is requiring Sarepta to conduct a postmarketing observational study to further assess the risk of serious liver injury. The study will enroll approximately 200 patients with DMD and follow them for at least 12 months after administration of Elevidys, with periodic liver function assessments.

Sarepta officials said they expect to quickly commence a study of an enhanced sirolimus immunosuppressive regimen to address the risk of ALI and ALF so that, with the concurrence of the FDA, dosing may resume for non-ambulatory patients.