Understanding eligibility

Who can receive ELEVIDYS

Many families have questions about whether ELEVIDYS is an appropriate treatment. One of the first steps is to consider eligibility. Below are answers to common questions about who may receive ELEVIDYS.

Who is eligible for ELEVIDYS?

ELEVIDYS is approved for people with Duchenne aged 4 years and up, regardless of ambulation. Use in non-ambulatory people was approved under accelerated approval, which allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit.

Treatment with ELEVIDYS increased the marker, ELEVIDYS micro-dystrophin. There were a limited number of participants who were non-ambulatory (8), and the impact on motor function was not studied in non-ambulatory people.

Verification of clinical benefit may be needed for ELEVIDYS to continue to be approved for non-ambulatory people with Duchenne. Studies are ongoing to assess this potential impact.

Who determines eligibility?

A doctor at a treatment center that administers ELEVIDYS will confirm eligibility.

What genetic mutations may be eligible?

All mutations except for deletions in exons 8 and/or 9 are eligible. Note: duplication mutations in exons 8 and/or 9 may be eligible.

People with certain mutation deletions (in exons 1 to 17 and/or exons 59 to 71) may be at risk for an immune response affecting muscles (immune-mediated myositis).

Talk to your doctor about your genetic test results.

How is eligibility confirmed?

A genetic test is required to confirm a mutation in the dystrophin gene. If you’ve had a test already, provide the report to your doctor to see if an updated test may be needed.

An antibody test is required to measure preexisting antibodies to the ELEVIDYS vector, called AAVrh74. If the levels are too high, ELEVIDYS may not be an option.

Your doctor will perform additional tests to confirm ELEVIDYS is appropriate.

Why antibodies matter

Antibodies are an important part of the immune system. They’re proteins your body creates to protect you from things like viruses. When a virus enters the body, the immune system gets to work, making antibodies to fight it. Once the virus is gone, the antibodies stay in your body, ready to attack that particular virus if it appears again.

VECTOR

Gene therapies, like ELEVIDYS, use vectors as the delivery mechanism for gene therapy components. Vectors are deactivated viruses that have been changed in a lab to prevent them from multiplying or causing an illness.

If someone has previously been exposed to a virus that is similar to a gene therapy vector, antibodies will be waiting to attack it. This will prevent the gene therapy from working and could cause harmful side effects. This is why antibody testing is part of the eligibility process; if levels are too high, treatment may not be an option.

Antibody testing is part of the elegibiliy process

Next up: Getting ready for treatment

Now that you’ve seen who may be eligible for ELEVIDYS, learn more about what to expect before the infusion.

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).