What is Elevidys® and how does it work?
Elevidys® (delandistrogene moxeparvovec-rokl) is a one-time gene therapy treatment for Duchenne muscular dystrophy (DMD), made by the pharmaceutical company Sarepta. Approved by the Food and Drug Administration (FDA) in 2023, Elevidys is designed to strengthen muscles by delivering a shortened version of the dystrophin gene, known as micro-dystrophin. This compensates for the missing or broken dystrophin gene that is the cause of DMD.
To deliver the micro-dystrophin gene, Elevidys uses a “vector” made from a harmless virus called AAVrh74 that is good at getting inside of cells. Once infused into the bloodstream, Elevidys gets into muscle cells, prompting them to produce the micro-dystrophin protein. This protein helps protect muscle cells and prevent them from degenerating.
Boston Children’s is a designated Center of Excellence for Elevidys treatment.
Is my child eligible for Elevidys?
Elevidys is approved for boys/men ages 4 and older who have had a genetic test confirming that they have DMD. Eligible individuals must have an antibody test to ensure that they do not already have antibodies against Elevidys, and have a medical assessment to ensure they don't have any other health conditions that would affect their ability to undergo treatment.
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What does Elevidys gene therapy entail?
Elevidys is given as a single intravenous (IV) infusion in Boston Children’s outpatient infusion clinic. After the infusion, which takes one to two hours, children stay in the infusion clinic for at least three hours for observation. The entire visit is approximately eight hours, between preparation, the infusion, and observation after the infusion.
Boys receiving Elevidys will need to take a higher dose of corticosteroid medication for at least three months after the infusion. This is meant to reduce the risk of liver inflammation and liver injury, which sometime occurs as a side effect of Elevidys treatment.
Before receiving Elevidys, your child should be up to date on his vaccinations, since the high-dose corticosteroid may affect what vaccines are safe to give. Vaccinations should be completed at least four weeks before Elevidys gene therapy.
After the treatment, families are asked to stay close to the hospital for two or three months and return for weekly blood work and monitoring. Children also have regular checkups with neuromuscular specialists and other clinicians in Neuromuscular Center.
Is Elevidys a cure for DMD?
Elevidys is not expected to cure DMD, but the FDA granted accelerated approval based on clinical trials suggesting that it is likely to increase muscle strength and mobility. Early test results indicate that boys receiving Elevidys do better on tests that ask them to stand up from a lying-down position or walk for 10 meters (about 33 feet). More information will become available as clinical trials continue.
Is Elevidys safe?
Clinical trials have found Elevidys to be largely safe. In clinical trials, the most common side effects were vomiting, nausea, fever, low platelet counts, and abnormalities on liver function tests. Most of these side effects appear during the first two weeks after treatment.
We do monitor children closely for several less-common side effects. These include:
- Inflammation (swelling) of the liver or increased risk of injury to the liver. Our medical team will monitor liver enzyme levels and may adjust the dose of corticosteroid medicine to lower this risk.
- Inflammation of the heart muscle (myocarditis), causing fatigue, dizziness, chest pain, and/or shortness of breath
- Inflammation of the muscle tissue (myositis), causing muscle pain and weakness
- Thrombocytopenia, or a low number of platelets in the blood, which can cause bruising or more bleeding than normal.
In addition, because of the higher steroid doses given after the infusion, children may be more susceptible to infections. For that reason, we suggest staying on alert for signs of illness and keeping children away from people with symptoms like a cold, coughing, fever, or flu-like symptoms while they are receiving high-dose steroids.
Will my health insurance cover Elevidys?
Because Elevidys is still new, it’s not yet clear to what extent insurance providers will cover this therapy. We encourage you to talk with your health insurance company and medical team. Boston Children’s Financial Services staff will guide you through the insurance approval process. In addition, Sarepta, the manufacturer of Elevidys, has a program called SareptAssist, which can assist with exploring insurance benefits and financial assistance options.
Questions?
Contact the Neuromuscular Center:
- U.S.: 617-355-6388
- International: +1-617-355-5209
