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 Aug 10, 2023.

What is Elevidys?

Elevidys is a recombinant gene therapy for Duchenne muscular dystrophy (DMD). Elevidys makes it possible for the body to make a protein (Elevidys micro-dystrophin) that contains copies of important sections of dystrophin proteins that were defective in the muscles of patients with Duchenne muscular dystrophy.

Elevidy is given as a single intravenous dose to Duchenne muscular dystrophy patients that are 4 to 5 years old and do not have contraindications to treatment.

Elevidys (delandistrogene moxeparvovec-rokl) received FDA approval on June 22, 2023, through the Accelerated Approval Pathway.

How does Elevidys work?

In Duchenne muscular dystrophy the defective gene means that 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, it is hoped this will 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.

Who can use Elevidys?

Elevidys is FDA-approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.

Patients should have anti-AAVrh74 total binding antibody titers <1:400. An FDA-authorized test for the detection of AAVrh74 total binding antibodies is not currently available. 

Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

Patient’s 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 

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, which is 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 as a result of 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?

The most common Elevidys side effects in clinical studies were: 

• vomiting
• nausea
• liver function test increased
• pyrexia
• thrombocytopenia.

The following are clinically significant adverse reactions

• acute serious liver injury
• immune-mediated myositis
• myocarditis.

Warnings

Acute Serious Liver Injury has been observed when using Elevidys. Before using this medicine liver function test should be performed, then monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 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 swallowing difficulty, difficulty breathing or a weak voice, as these may be symptoms of myositis.

Myocarditis 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. 

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 a 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.

Dosing information

The dose of Elevidys is based on body weight.
The recommended dose is 1.33 × 1014 vector genomes per kilogram (vg/kg) of body weight (or 10 mL/kg body weight).

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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