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 Apr 11, 2025.

What is Elevidys?

Elevidys is a gene therapy used for Duchenne muscular dystrophy (DMD) to improve motor function and strengthen muscles.

Elevidys works by helping the body produce micro-dystrophin - a smaller version of dystrophin, a crucial protein that supports muscle health and is lacking in individuals with Duchenne muscular dystrophy.

Elevidys is a one-time gene therapy administered as an intravenous infusion.

Elevidys FDA approval

Elevidys is FDA-approved to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene and are 4 years older. Elevidys has a traditional FDA approval for ambulatory patients and an accelerated approval for non-ambulatory patients.

Elevidys should not be used in patients with a deletion in exon 8 and/or exon 9 in the DMD gene. It should also not be used if you have elevated levels of antibodies to the Elevidys vector (AAVrh74).

Elevidys FDA approval was granted to Sarepta Therapeutics on June 22, 2023.

How does Elevidys work?

Elevidys mechanism of action is by providing a gene with instructions for the body to make a shorter version of dystrophin called micro-dystrophin which prevents muscle damage and helps strengthen muscles. 

Elevidys is a gene therapy that contains 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, is hoped to improve motor function.  A clinical benefit of Elevidys has not been established as it is still being studied whether it will improve physical function and mobility in ambulatory patients.

Elevidys is an adeno-associated virus vector-based gene therapy

What is Duchenne Muscular Dystrophy?

People with Duchenne muscular dystrophy have a faulty gene that prevents their bodies from producing dystrophin - a protein essential for healthy muscle cells. Without dystrophin, muscles become damaged over time, leading to a loss of strength. This makes it harder to perform everyday tasks and activities, and eventually, it can affect vital body functions.

What are the side effects of Elevidys?

Common Elevidys side effects were vomiting, nausea, increased fever, and low blood platelet levels.

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 Elevidys infusion.

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. Elevidys 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 Elevidys?

• Elevidys is a single-dose (one-time treatment) given as an intravenous infusion.
• Before the Elevidys infusion, 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

• Elevidys is shipped and delivered at ≤ −60ºC [−76ºF].
• Elevidys 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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