Elevidys

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, 2024.

What is Elevidys?

Elevidys is a gene therapy for Duchenne muscular dystrophy (DMD) that may strengthen muscles by helping the body make a protein called micro-dystrophin. This helps protect muscle cells and prevent them from degenerating. Elevidys is a one-time treatment given to patients as a single intravenous dose. 

Elevidys FDA approval was received on June 22, 2023, for the company Sarepta Therapeutics. Elevidys is FDA-approved 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 older.

If you have elevated levels of antibodies to the Elevidys vector (AAVrh74), you are not eligible for treatment with this medicine. Elevidys can not be used 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.

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, 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.

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.

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.

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. 

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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