Elevidys (delandistrogene moxeparvovec)
An approved treatment for Duchenne Muscular Dystrophy.
The same compound appears under different names depending on the context. Here is how to identify Delandistrogene moxeparvovec wherever you encounter it, plus the key facts at a glance.
- Generic name
- Delandistrogene moxeparvovec
- Brand name
- Elevidys
- Development code
- SRP-9001
- Drug class
- Gene therapy
- Manufacturer
- Sarepta Therapeutics
- How it's taken
- Given as a single one-time intravenous infusion.
The first gene therapy approved for Duchenne muscular dystrophy. A single IV infusion delivers a functional mini-version of the dystrophin gene to muscle cells, aiming to slow or halt disease progression.
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Where Delandistrogene moxeparvovec fits
First gene therapy for DMD, delivering a shortened but functional dystrophin gene. One-time IV infusion for ambulatory patients, aiming to produce the missing protein rather than skip around mutations.
How Delandistrogene moxeparvovec works
Elevidys uses a harmless virus (AAV) to deliver a shortened but working copy of the dystrophin gene directly into muscle cells. In DMD, the full dystrophin gene is broken, so muscles lack the protein they need to stay strong. The mini-dystrophin produced by Elevidys acts as a structural support inside muscle fibers, helping protect them from damage during movement.
Mechanism: AAV-based gene therapy delivering a shortened but functional dystrophin gene (micro-dystrophin)
Side effects and safety
Carries a Boxed Warning for acute serious liver injury and acute liver failure, including fatal outcomes. The most common side effects are vomiting and nausea, liver injury, fever, low platelet counts, and a rise in troponin-I (a marker of heart muscle injury). Liver tests are needed before treatment and weekly for the first 3 months after. Platelets are checked weekly for the first 2 weeks and troponin-I weekly for the first month. The steroids given with Elevidys raise the risk of serious infections, and immune reactions, including serious muscle inflammation (myositis) in some mutation types, can occur. Serious, life-threatening inflammation of the heart muscle (myocarditis) can happen, from within a day to more than a year after treatment. Infusion reactions, including severe allergic reactions (anaphylaxis), can occur during or hours after the infusion, so patients are watched for at least 3 hours afterward. Families should stay close to an appropriate hospital or clinic for at least 2 months after the infusion.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Delandistrogene moxeparvovec
Given as a single one-time intravenous infusion. Patients receive corticosteroids before and after infusion to manage immune response. Treatment is administered at specialized centers.
Availability and cost
Only available as the brand-name product.
One-time AAV gene therapy delivering a micro-dystrophin gene. Complex viral vector manufacturing and one-time curative-intent dosing for a rare genetic disease.
Help paying for Elevidys
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Insurance and case manager help
SareptAssist team helps with treatment logistics and questions before, during and after the one-time ELEVIDYS infusion.
The official page does not say who qualifies. Ask the program. · source - Copay help
Co-pay Assistance Program may help commercially insured patients with co-pays, co-insurance and deductibles. Not for government insurance like Medicare.
For: private insurance · source
Good to know: Call 1-888-SAREPTA (Mon-Fri 8:30am-6:30pm ET); only for US patients prescribed ELEVIDYS. Copay terms come from Sarepta's corporate SareptAssist page ('a Sarepta product'). The ELEVIDYS page does not describe a free-drug program. Patient Education Liaisons: 1-888-848-6374.
- From a charity · NORD RareCareDuchenne Muscular Dystrophy Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · NORD RareCareDuchenne Muscular Dystrophy Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · The Assistance FundDuchenne Muscular Dystrophy fundOpen
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “OPEN — Accepting New Patients. TAF is currently accepting new patient enrollments for this program.” - From a charity · Muscular Dystrophy AssociationMDA Durable Medical Equipment (DME) Grant Program fundApply directly
Pays for: Medical equipment (wheelchairs, lifts, canes and other DME), up to $1,000 per year.
The foundation says: “Status not shown on page”
Access and eligibility
Elevidys is not suitable for patients with deletions involving exon 8 and/or exon 9 of the DMD gene. Patients must also not have high levels of anti-AAVrh74 antibodies. The label also says Elevidys is not recommended for people with existing liver problems or an active liver virus infection, or for anyone who got a vaccine or had an infection in the past 4 weeks.
Source: Elevidys.com (Sarepta Therapeutics)
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
Initially approved June 2023 under accelerated approval for ambulatory patients ages 4-5. In June 2024 it received full (traditional) approval for ambulatory patients 4 and older, and accelerated approval was added for non-ambulatory patients.
Following two reports of fatal acute liver failure in non-ambulatory patients in 2025, the FDA added a Boxed Warning and revised the indication to limit treatment to ambulatory patients age 4 and older. Three-year EMBARK data (January 2026) showed 70%+ reduction in decline rate versus external controls.
Development history
Developed by Sarepta Therapeutics, Elevidys was the first gene therapy approved for DMD. Initial accelerated approval in 2023 was based on micro-dystrophin expression.
The label was expanded in 2024 to non-ambulatory patients but then restricted again in 2025 after the FDA approved a Boxed Warning for acute liver injury and limited the indication to ambulatory patients only. Sarepta is now studying an immunosuppressive regimen aimed at restoring access for non-ambulatory patients.
Explore Duchenne Muscular Dystrophy trials
Other Duchenne Muscular Dystrophy treatments
Common questions about Delandistrogene moxeparvovec
▸What is Delandistrogene moxeparvovec (Elevidys)?
The first gene therapy approved for Duchenne muscular dystrophy. A single IV infusion delivers a functional mini-version of the dystrophin gene to muscle cells, aiming to slow or halt disease progression.
▸How does Delandistrogene moxeparvovec work?
Elevidys uses a harmless virus (AAV) to deliver a shortened but working copy of the dystrophin gene directly into muscle cells. In DMD, the full dystrophin gene is broken, so muscles lack the protein they need to stay strong. The mini-dystrophin produced by Elevidys acts as a structural support inside muscle fibers, helping protect them from damage during movement.
▸What are the side effects of Delandistrogene moxeparvovec?
Carries a Boxed Warning for acute serious liver injury and acute liver failure, including fatal outcomes. The most common side effects are vomiting and nausea, liver injury, fever, low platelet counts, and a rise in troponin-I (a marker of heart muscle injury). Liver tests are needed before treatment and weekly for the first 3 months after. Platelets are checked weekly for the first 2 weeks and troponin-I weekly for the first month. The steroids given with Elevidys raise the risk of serious infections, and immune reactions, including serious muscle inflammation (myositis) in some mutation types, can occur. Serious, life-threatening inflammation of the heart muscle (myocarditis) can happen, from within a day to more than a year after treatment. Infusion reactions, including severe allergic reactions (anaphylaxis), can occur during or hours after the infusion, so patients are watched for at least 3 hours afterward. Families should stay close to an appropriate hospital or clinic for at least 2 months after the infusion.
▸How is Delandistrogene moxeparvovec taken?
Given as a single one-time intravenous infusion. Patients receive corticosteroids before and after infusion to manage immune response. Treatment is administered at specialized centers.
▸Is Delandistrogene moxeparvovec FDA approved?
Yes, Delandistrogene moxeparvovec (Elevidys) is FDA approved (2023) for the treatment of Duchenne Muscular Dystrophy.
▸What is the boxed warning about liver injury?
Elevidys carries a Boxed Warning for acute serious liver injury, including acute liver failure and fatal outcomes. Two non-ambulatory patients died from acute liver failure after receiving Elevidys. The risk appears higher in non-ambulatory patients. Because of this, the FDA restricted the indication to ambulatory patients only in 2025 and requires liver function monitoring before and after treatment. Patients and families should discuss the liver risks in detail with their physician before proceeding.
▸Why was the label restricted to ambulatory patients only?
After the initial approval included both ambulatory and non-ambulatory patients, two deaths from acute liver failure occurred in non-ambulatory patients in 2025. The FDA concluded that the benefit-risk balance was unfavorable for non-ambulatory patients given the liver injury risk and revised the label to limit Elevidys to ambulatory patients aged 4 and older. Sarepta is studying modified immunosuppressive protocols that could potentially restore access for non-ambulatory patients in the future.
▸What is micro-dystrophin?
The full dystrophin gene is too large to fit inside an AAV delivery vehicle (the virus used to carry the gene into cells). Micro-dystrophin is a shortened version of the gene that retains the most critical functional domains. The protein it produces is smaller than normal dystrophin but can still provide structural support to muscle fibers. This concept is inspired by Becker muscular dystrophy, where patients naturally produce shorter-than-normal dystrophin and have a milder disease course than DMD.
▸Can a patient receive Elevidys more than once?
No. Elevidys is designed as a one-time treatment. After the first infusion, the body develops antibodies against the AAVrh74 viral vector, which would neutralize a second dose. Patients with pre-existing high levels of anti-AAVrh74 antibodies are also ineligible for the initial treatment. This is a fundamental limitation shared by all current AAV-based gene therapies.
▸What does the 3-year EMBARK data show?
Three-year follow-up data from the EMBARK trial, reported in January 2026, showed a 70% or greater reduction in the rate of functional decline compared to external control groups of untreated DMD patients. This long-term data supports the durability of micro-dystrophin expression and suggests sustained clinical benefit over multiple years, though ongoing monitoring continues.
▸Which mutations are not eligible for Elevidys?
Patients with deletions involving exon 8 and/or exon 9 of the DMD gene are not eligible for Elevidys because the micro-dystrophin construct may not function properly in these patients. Additionally, patients with high pre-existing antibodies to the AAVrh74 viral vector cannot receive the therapy. Genetic testing and antibody screening are required before treatment.