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ELEVIDYS directly addresses the missing dystrophin in the body

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Genetic mutation that causes Duchenne

Duchenne is caused by a change that happens in the dystrophin gene before birth. This change stops the body from making dystrophin, a protein needed for muscles to work properly.

Without dystrophin, muscles become irreversibly damaged. Over time, this leads to ongoing loss of muscle strength, making it difficult to walk and perform everyday activities.

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Early intervention may help slow muscle damage. Talk to your doctor about a treatment plan that includes therapy to increase dystrophin

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ELEVIDYS delivers a new version of dystrophin to the body

ELEVIDYS delivers a new version of dystrophin

ELEVIDYS instructs the body to make a new, shorter version of dystrophin, called ELEVIDYS micro-dystrophin. It’s not exactly the same as the body’s natural dystrophin, but it helps protect muscles from damage.

See how ELEVIDYS works in the body

How ELEVIDYS works video
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The science behind ELEVIDYS

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.

 

Duchenne muscle cell without treatment

 

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.

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.

Two important parts of ELEVIDYS include:

ELEVIDYS vector

ELEVIDYS vector, which carries the transgene to muscle cells

ELEVIDYS transgene

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.

 

ELEVIDYS transgene in the muscle cell

 

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”

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Next up: See how ELEVIDYS was studied

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

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Colton received ELEVIDYS in 2024

What is ELEVIDYS?

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 not recommended for individuals with:

  • Preexisting liver problems or liver infection because of the high risk of rapid serious liver injury and rapid liver failure
  • Recent vaccination (within 4 weeks of ELEVIDYS treatment)
  • Current or recent infections (within 4 weeks of ELEVIDYS treatment)
     

Important Safety Information

What is the most important information to know about ELEVIDYS?
Rapid Serious Liver Injury and Rapid Liver Failure

  • ELEVIDYS can increase certain liver lab test levels and cause rapid serious liver injury, rapid liver failure, and death. Patients with preexisting liver problems may be at higher risk.
  • Complication of blood clots in the blood vessel in the abdomen that helps carry blood from the intestines to the liver has happened.
  • Patients will receive oral corticosteroid medication before and after ELEVIDYS infusion and will need weekly blood tests to monitor liver function for 3 months or longer after treatment.
  • For at least 2 months following ELEVIDYS infusion, stay close to a healthcare facility that the doctor recommends.
  • Contact a doctor 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.

Serious Infection

  • Because patients will be taking corticosteroids as part of ELEVIDYS treatment, this may lower the ability of their immune system to fight infections and make it easier to get an infection. Getting an infection (like a cold, flu, stomach flu, ear infection, chest infection) before or after ELEVIDYS infusion could lead to more serious health problems, including death.
  • Contact a doctor right away if you notice any signs of infection, such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Vaccinations should be completed at least 4 weeks before starting the corticosteroids that are part of the ELEVIDYS treatment.
  • ELEVIDYS should not be given to patients with an infection.

Inflammation of the Heart Muscle

  • Serious and life-threatening inflammation of the heart muscle has happened following ELEVIDYS infusion.
  • Patients will need weekly blood tests for a heart protein that can detect damage to the heart muscle cells for the first month after treatment.
  • Contact a doctor right away if the patient begins to experience chest pain and/or trouble breathing or shortness of breath.

Infusion-related Reactions

  • Infusion-related reactions, including hypersensitivity and serious allergic reactions (anaphylaxis), have happened during and after ELEVIDYS infusion.
  • Contact a doctor right away if you notice: 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.

Immune Response Affecting Muscles (Immune-mediated Myositis)

  • Immune response affecting muscles, including serious and life-threatening reactions, has happened in patients about 1 month after receiving ELEVIDYS infusion.
  • Contact a doctor immediately if the patient experiences any unexplained increased muscle pain, tenderness, or weakness, including trouble swallowing, breathing, or speaking.

Antibodies to ELEVIDYS

  • Patients need to have blood tests to ensure that they do not have antibodies that may prevent them from being able to receive ELEVIDYS. High levels of antibodies may keep the medicine from working as intended.
  • Treatment with ELEVIDYS is not recommended for patients who have high antibodies to the vector, the part of gene therapy used to deliver ELEVIDYS.

You should discuss the potential benefits and risks of ELEVIDYS with your doctor.

Who should not receive ELEVIDYS?
Individuals with a certain type of genetic mutation, called a deletion, involving any portion of or the entire exon 8 and/or exon 9 in the DMD gene, should not receive ELEVIDYS.

Are there any considerations for vaccination schedules and ELEVIDYS?
Patient vaccinations should be up to date with current immunization guidelines. Vaccinations should be completed at least 4 weeks before starting corticosteroids that are part of the ELEVIDYS treatment.

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 most common side effects of ELEVIDYS?
The most common side effects that occurred in patients treated with ELEVIDYS were vomiting, nausea, liver injury, fever, lower number of platelets (a kind of blood cell that helps you stop bleeding), and higher levels of heart protein that can detect damage to muscle cells in the heart. 

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).

Please see full Prescribing Information, including Boxed Warning, and Medication Guide.