Generic name: delandistrogene moxeparvovec-rokl
Dosage form: suspension for intravenous infusion
Drug class: Miscellaneous uncategorized agents

Medically reviewed by Melisa Puckey, BPharm. Last updated on Jun 24, 2025.

What is Elevidys?

Elevidys is a gene therapy designed to treat Duchenne muscular dystrophy (DMD). Elevidys works by helping the body produce a protein called micro-dystrophin, which may strengthen muscles and improve motor function.

Elevidys is given as a one-time intravenous (IV) infusion, meaning patients receive a single dose through a vein.

Elevidys FDA approval is to treat Duchenne muscular dystrophy (DMD) in patients who are ambulatory (traditional approval) or non-ambulatory (accelerated approval), have a confirmed mutation in the DMD gene, and are 4 years or older. FDA approval was first granted to Sarepta Therapeutics on June 22, 2023. 

This medicine should not be used if you have elevated levels of antibodies to the Elevidys vector (AAVrh74) or if you have any deletion in exon 8 and/or exon 9 in the DMD gene.

How does Elevidys work?

Elevidys helps the body make a protein (micro-dystrophin) that prevents muscle cells from degenerating, which may help strengthen muscles and improve motor function.

In Duchenne muscular dystrophy, the defective gene means the dystrophin protein does not function properly. Elevidys is a gene therapy that introduces the code or instructions on how to make the most important sections of the dystrophin protein. It does not make the whole protein, as the code is too large.  The introduced code makes microdystrophin, which contains selected sections of dystrophin protein present in normal muscle cells, and is hoped to improve motor function.  A clinical benefit of this medicine has not been established, as it is still being studied whether it will improve physical function and mobility.

Elevidys mechanism of action is as an adeno-associated virus vector-based gene therapy

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy is a serious but rare genetic condition that becomes worse over time, leading to weakness and wasting away of the body’s muscles. The cause of Duchenne muscular dystrophy is a defective gene that codes for the dystrophin protein, an important component of muscle cells.

The lack of dystrophin causes symptoms such as trouble walking and running, falling frequently, fatigue, learning disabilities/difficulties, heart issues due to the impact on the heart muscle, and breathing problems due to weakness of respiratory muscles involved in breathing.

Symptoms typically begin in childhood, often between 3 to 6 years old, and as the disease progresses, life-threatening heart and respiratory problems can occur. Disease severity and life expectancy do vary, but patients often succumb to the disease in their 20s or 30s because of heart and/or respiratory failure.

Elevidys side effects

Common Elevidys side effects were vomiting (65%), nausea (43%), liver injury (40%), fever (28%), and low blood platelet levels (8%). These were the side effects that affected  5% or more patients in clinical studies.

The following are clinically significant Elevidys side effects:

• acute serious liver injury
• immune-mediated inflammation of muscles (myositis)
• inflammation of the heart (myocarditis)
• hypersensitivity reactions and anaphylaxis
• infections.

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Warnings

Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following infusion. Patients are closely monitored during and for at least 3 hours after the end of infusion for signs and symptoms of infusion-related reactions, including tachycardia, tachypnea, lip swelling, difficulty breathing, nasal flaring, urticaria, flushing, lip pruritus, rash, cheilitis, vomiting, nausea, rigors, and pyrexia.

Acute serious liver injury has been observed when using Elevidys. Before using this medicine, a liver function test should be performed. Then, liver function (clinical exam, GGT, and total bilirubin) should be monitored weekly for the first three months following infusion.  Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone the infusion administration in patients with acute liver disease until resolved or controlled. Systemic corticosteroid treatment is recommended for patients before and after Elevidys infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended.

Immune-mediated myositis (an immune response affecting muscles). Patients should contact their healthcare provider immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, difficulty breathing, or a weak voice, as these may be myositis symptoms.

Acute serious myocarditis (inflammation of the heart) and troponin-I elevations have been observed following Elevidys infusion in clinical trials. Patients should contact their healthcare provider immediately if they experience chest pain and/or shortness of breath. 

Pre-existing Immunity against AAVrh74 In AAV-vector based gene therapies. Preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with this infusion, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies before infusion. This infusion should not be administered in patients with elevated anti-AAVrh74 total binding antibody titers (≥1:400)

Infections. While taking corticosteroids, an infection before or after the Elevidys infusion could lead to more serious complications. Contact a healthcare provider immediately if symptoms suggestive of infection are observed (e.g., coughing, wheezing, sneezing, runny nose, sore throat, or fever). 

How will I receive this medicine?

• Elevidys is a single-dose (one-time treatment) given as an intravenous infusion.
• Before the infusion of this medicine, you will have your liver function assessed and other blood tests.
• You should not have an infusion while you have an infection.
• To reduce the risk associated with an immune response, you may be prescribed corticosteroids starting one day before the infusion.

Patients’ immunizations should be up-to-date with current immunization guidelines before starting the corticosteroid regimen required for Elevidys infusion. Vaccinations should be completed at least 4 weeks prior to initiation of the corticosteroid regimen. 

Dosing information

The dose of Elevidys is based on body weight.

Patients weighing 10 to 70 kg: The recommended dose is 1.33 × 1014 vector genomes per kilogram (vg/kg) of body weight (or 10 mL/kg body weight).

Patients 70 kg or greater: Maximum dose of 9.31 × 1015 vg total fixed dose.

Interactions

Before starting the corticosteroid regimen required before Elevidys infusion, consider the patient’s vaccination status. Patients should, if possible, be brought up-to-date with all immunizations in current immunization guidelines. Vaccinations should be completed at least 4 weeks before starting the corticosteroid regimen. 

Elevidys Package Insert 

HCPs and patients often use the Elevidys Package Insert (PI) for more detailed information about this medicine. The Elevidys Package Insert (prescribing information) contains more comprehensive information on Indications and Usage, Dosage and Administration, Clinical Pharmacology, Clinical Studies, Drug Interaction, and more. Discuss any medical questions you have with your HCP (health care professional). This is not all the information you need to know about this medicine for safe and effective use, and it does not take the place of talking to your doctor about your treatment.

The Package Insert is sometimes called Elevidys Prescribing Information (PI) or FDA label.

Elevidys J code

Elevidys J code is J1413.
J codes are used for medicines that are not taken orally and include injections, inhalations, and chemotherapies. J codes are important for accurate and consistent coding for billing and reimbursement purposes.

Your physician will need the Elevidys J code when filling out forms for your treatment.

Storage

• Infusion is shipped and delivered at ≤ −60ºC [−76ºF].
• Infusion can be refrigerated for up to 14 days when stored at 2°C to 8°C (36º F to 46º F) in the upright position. 
• Do not refreeze.
• Do not shake.
• Do not place back in the refrigerator once brought to room temperature.
• Follow local guidelines on handling biological waste.

Manufacturer

Manufactured for: Sarepta Therapeutics, Inc. Cambridge, MA 02142 USA

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Further information

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