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Crescent

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How ELEVIDYS works

In Duchenne, a genetic mutation, or change, in the dystrophin gene prevents the body from making dystrophin. Without dystrophin, muscles become damaged and, over time, people with Duchenne lose muscle strength and the ability to perform everyday activities.

So how does ELEVIDYS help?

ELEVIDYS delivers a gene that instructs the body to make a new, shorter version of dystrophin.

See ELEVIDYS in action

Watch this video to learn how ELEVIDYS is intended to work in the body.

Take a closer look

Interested in a more detailed overview of the science behind ELEVIDYS?

First, let’s consider how muscles function. Muscles need an important protein called dystrophin. Dystrophin’s job is to help protect muscles from injury during everyday activities like standing up from a chair, walking, or climbing stairs.

But why do people with Duchenne have weaker muscles? They have a genetic mutation, or change, that prevents the body from making dystrophin.

In people with Duchenne, muscles become damaged beyond repair, resulting in the loss of muscle strength. Eventually, this will impact the ability to perform everyday activities.

Treatment that increases dystrophin is an important early step in a Duchenne treatment plan.

How could ELEVIDYS help with this lack of dystrophin? ELEVIDYS is designed to help the body make a new, shorter form of dystrophin called ELEVIDYS micro-dystrophin. ELEVIDYS micro-dystrophin is smaller than the body’s natural dystrophin, but it still has the key pieces needed to make a protein.

ELEVIDYS vector, which carries the transgene to muscle cells

ELEVIDYS transgene, which is the instructions that tell the body to make this new protein

The vector enters muscle cells to deliver the transgene:

ELEVIDYS vector entering the muscle cell

The vector then exits the muscles and is gradually eliminated from the body, while the transgene stays behind.

The transgene gets to work, providing the instructions to make ELEVIDYS micro-dystrophin. It only needs to be delivered once to do its job: once inside the body, the transgene provides the instructions needed to make ELEVIDYS micro-dystrophin.

Duchenne muscle cell with ELEVIDYS micro-dystrophin

INTERESTING FACT

The dystrophin gene is the largest known human gene. It’s so large that it can’t fit inside gene therapy vectors, including the one used in ELEVIDYS. The ELEVIDYS transgene was designed to be smaller, using select parts of natural dystrophin researchers believe are important so that it can fit within a vector. Since it’s smaller, the dystrophin it helps produce is also smaller — that’s why it’s called “micro-dystrophin.”

Next up: See how ELEVIDYS was studied

Now that you know how ELEVIDYS works, explore what was measured in the clinical trials.

Boy who received treatment with ELEVIDYS

What is ELEVIDYS (delandistrogene moxeparvovec-rokl)?

ELEVIDYS is a prescription gene therapy used to treat ambulatory individuals at least 4 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.

ELEVIDYS is approved under accelerated approval for non-ambulatory patients at least 4 years old with DMD who have a confirmed mutation in the DMD gene. Accelerated approval allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. ELEVIDYS treatment increased the marker, ELEVIDYS micro-dystrophin in skeletal muscle. Verification of a clinical benefit may be needed for ELEVIDYS to continue to be approved for non-ambulatory patients with DMD.

Important Safety Information

Who should not receive ELEVIDYS?
Individuals with certain types of mutations, any deletion in exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.

What is the most important information to know about ELEVIDYS?
Infusion-related reactions, including hypersensitivity and serious allergic reactions (anaphylaxis), have occurred during and after ELEVIDYS infusion. Symptoms may include fast heart rate, fast breathing, swollen lips, shortness of breath, nostrils widening, hives, red and blotchy skin, itchy or inflamed lips, rash, vomiting, nausea, chills, and fever. Your doctor will monitor you during and at least 3 hours after ELEVIDYS infusion. If an infusion-related reaction occurs, your doctor may slow or stop the ELEVIDYS infusion and provide additional medical treatment as needed. Contact a healthcare provider immediately if infusion-related symptoms occur.

ELEVIDYS can increase certain liver enzyme levels and cause acute serious liver injury. Patients will receive oral corticosteroid medication before and after infusion with ELEVIDYS and will undergo weekly blood tests to monitor liver enzyme levels for 3 months after treatment. Contact a healthcare provider immediately if the patient’s skin and/or whites of the eyes appear yellowish or if the patient misses a dose of corticosteroid or vomits it up.

Administration of ELEVIDYS may be delayed in patients who have acute liver disease until the condition is resolved or under control. Patients with preexisting liver impairment, chronic liver infection, or acute liver disease may be at higher risk of acute serious liver injury.

Immune-mediated myositis (an immune response affecting muscles) was observed in patients with a deletion mutation in the DMD gene that is contraindicated. Patients with certain mutation deletions (in exons 1 to 17 and/or exons 59 to 71) may be at risk for a severe immune-mediated myositis reaction. Caregivers should contact a healthcare provider immediately if the patient experiences any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, breathing, or speaking, as these may be symptoms of myositis.

Myocarditis (inflammation of the heart) has been observed within days following ELEVIDYS infusion. The patient’s doctor will conduct weekly blood tests for the first month after treatment to evaluate troponin-I (a cardiac protein that can detect damage to muscle cells in the heart). Caregivers should contact a healthcare provider immediately if the patient begins to experience chest pain and/or shortness of breath. More frequent monitoring may be required if the patient has cardiac symptoms.

Patients need to have blood tests to ensure that they do not have antibodies that may prevent them from being able to receive ELEVIDYS, as introducing the gene therapy could increase the risk of a severe allergic reaction or prevent desired therapeutic levels. Treatment with ELEVIDYS is not recommended for patients who have high antibodies to the vector, the part of gene therapy used to deliver ELEVIDYS.

Due to the need to follow a corticosteroid regimen, an infection (such as cold, flu, gastroenteritis [stomach flu], otitis media [ear infection], bronchiolitis [respiratory infection], etc) before or after ELEVIDYS infusion could lead to more serious complications. Caregivers should contact a healthcare provider immediately if they see any symptoms suggestive of infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.

Are there any considerations for vaccination schedules and ELEVIDYS?
Patient vaccinations should be up to date with current immunization guidelines. Vaccinations should be received at least 4 weeks prior to starting the corticosteroid regimen that is required before receiving ELEVIDYS.

Are there any precautions that need to be considered when handling a patient’s bodily waste?
Vector shedding of ELEVIDYS occurs primarily through body waste. Patients and caregivers should use proper hand hygiene, such as hand washing when coming into direct contact with patient body waste. Place potentially contaminated materials that may have the patient’s bodily fluids/waste in a sealable bag and dispose into regular trash. Precautions should be followed for 1 month after ELEVIDYS infusion.

What are the possible or likely side effects of ELEVIDYS?
The most common side effects that occurred in patients treated with ELEVIDYS were vomiting, nausea, liver injury, fever, and decreased platelet counts.

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).