by: Parent Project Muscular Dystrophy
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.
Read the FDA’s press release here.
Read Sarepta’s community letter:
Dear Duchenne community,
Recently, we shared the announcement that the prescribing information (also known as “the label”) for ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy (DMD) has been updated. With this letter, we want to share what has changed, provide the MedicationGuide, answer questions about what it means for families and patients interested in pursuing treatment, and reaffirm our commitment to ensuring patient safety, which is our highest priority.
What is a label change?
The prescribing information (PI) label is a living document that provides instructions and essential scientific information to healthcare professionals for the safe and effective use of a medicine. Often times, especially in rare disease, as real-world experience with a prescribed product grows post- approval, updates to the label are made to reflect new learnings around patients’ experiences with the therapy, including those related to adverse events.
Sarepta initiated the labeling update process for ELEVIDYS following the passing of a non-ambulatory patient from acute liver failure following treatment with ELEVIDYS, and has been working with FDA over the last several months to make updates to the labeling.
The revised labeling can be accessed PILINK as well as the MedicationGuide.
What changed with the ELEVIDYS label?
As previously shared, we voluntarily suspended commercial shipments of ELEVIDYS for non-ambulatory patients in June, while we pursued plans for an additional study of an enhanced immunosuppressive regimen in non-ambulatory patients.
At this time, the FDA review of the updated label is complete. Here we share key updates to the ELEVIDYS label:
• A boxed warning for the risk of acute serious liver injury (ALI) and acute liver failure (ALF). A boxed warning is meant by FDA to highlight certain contraindications or serious warnings and to call prominent attention to healthcare providers about potential adverse reactions and safe use instructions.
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- Removal of language regarding the use of ELEVIDYS for non-ambulatory patients. These measures aim to ensure the safe administration of ELEVIDYS in ambulatory patients while equipping healthcare providers, patients, and families with the scientific information needed to support informed, individualized treatment discussions.
- Expanded guidance for prescribers, including a modified pre- and post-infusion oral corticosteroids regimen and enhanced monitoring recommendations on a weekly basis for 3 months post-infusion to help ensure patients receiving ELEVIDYS have a standardized pre- and post-infusion treatment and monitoring experience.
- A new warning in the Warnings & Precautions section regarding increased susceptibility for serious infections due to immunosuppression.
Our commitment to non-ambulatory Duchenne patients remains unwavering. This label update allows us the time to study the use of enhanced immunosuppression therapy in non-ambulatory patients such that we will be able to have a more informed, evidence-based discussion regarding the path to future access to ELEVIDYS by non-ambulatory patients.
We understand the urgency this group of patients and their families feel; we feel it, too. We will work diligently with regulators and healthcare professionals and, most importantly, you, the Duchenne community, to ensure that non-ambulatory patients have equal access to informed decision-making about their care and the benefit-risk profile of innovative treatment options and will continue to support the entire Duchenne community.
We encourage families to read the Medication Guide and to talk to their doctor for a full understanding of the risks and benefits on an individual patient level.
Sincerely,
Wendy Erler
Senior Vice President, Patient Affairs
