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Understanding eligibility

Understanding eligibility

Understanding elegibility; Austin received treatment with ELEVIDYS

Who can receive ELEVIDYS

Many families have questions about whether ELEVIDYS is right for them. One of the first things to consider is eligibility, since ELEVIDYS can only be given to certain people. If you are considering ELEVIDYS, talk to your doctor about ordering an antibody test to help determine if ELEVIDYS may be appropriate. 

Below are answers to common questions about who may receive ELEVIDYS.

Eligibility is based on a number of factors, and will be determined by your child’s doctor through a series of tests. 

ELEVIDYS is approved for ambulatory people with Duchenne who are at least 4 years old and have a confirmed mutation in the dystrophin gene. 

ELEVIDYS is not recommended for those with preexisting liver problems, recent vaccinations (within 4 weeks), or current/recent infections (within 4 weeks). 

ELEVIDYS cannot be used in anyone with a whole or partial deletion in exon 8 and/or exon 9. 

Your child’s doctor or a doctor at an ELEVIDYS treatment center can help you confirm eligibility.

Your child's doctor or a doctor at an ELEVIDYS treatment center can help you confirm eligibility.

All mutations except for whole or partial 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) have experienced for a serious and sometimes life-threatening immune response affecting muscles (immune-mediated myositis).

Talk to your doctor about your genetic test results and how they may impact your child’s eligibility.

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 also 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 and ask you questions about recent vaccinations and infections to confirm whether 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.

Vectors work as the delivery mechanism for gene therapies

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
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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.

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Thomas received treatment with ELEVIDYS
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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)
     

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

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.