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Caring for your child after treatment

Caring for your child after treatment

Caring for your child after treatment; Thomas received treatment with ELEVIDYS

ELEVIDYS is a one-time treatment with a safety monitoring plan specifically designed to offer ongoing support after treatment. 

Your child will need to remain close to an appropriate healthcare facility for at least 2 months after treatment. You can work with your doctor to find the right facility for this ongoing monitoring. 

In clinical trials, most side effects occurred within 2 months of treatment, but weekly monitoring will continue for at least 3 months. Longer or more frequent monitoring may be needed, depending on your child’s test results. The doctor will order blood tests to check:

  • Liver function (weekly for the first 3 months)
  • Platelet counts (weekly for the first 2 weeks)
  • Troponin-I levels (weekly for the first month)
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  1. Treatment Day
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    Treatment Day

    The care team at the treatment center will closely watch for any infusion-related reactions during and for at least 3 hours after treatment

  2. Side effects possible during the first two weeks
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    Side effects possible during the first two weeks

    The most common side effects, which typically occur in the first 2 weeks, include vomiting (as early as on infusion day), nausea, fever, and low platelet count

  3. Side effects possible during the first month
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    Side effects possible during the first month

    Side effects that could occur within the first month include:

    • Inflammation of the heart (myocarditis)
    • Increase in troponin-I, a cardiac protein that can indicate damage to muscle cells in the heart
  4. Side effects possible during the first two months
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    Side effects possible during the first two months

    Side effects that could occur within the first 2 months include:

    • An immune response affecting muscles (immune-mediated myositis)
    • Liver injury
    To help reduce the risk of an immune response, your child will need to take oral treatment-related steroids. These will start shortly before the infusion day and will continue for at least 2 months.*
  5. Side effects possible during the first three months
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    Side effects possible during the first three months

    Liver function will continue to be monitored for at least 3 months

*Your doctor may adjust this length of time, the steroid dose, or how it’s given (oral vs intravenous) based on the person’s response to ELEVIDYS, which may include addressing any changes in liver function tests. It’s important to follow your doctor’s advice on oral treatment-related steroids, including during the tapering period, to help avoid serious side effects. Report any side effects to your doctor immediately.

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Care team after treatment
Sarepta Support

Your ELEVIDYS support network after treatment includes:

  • The treatment center care team, who will conduct ongoing weekly safety monitoring for at least 3 months
  • Your child’s regular neurologist, who will continue to consult on your child’s care and support posttreatment follow-ups
  • Your SareptAssist Case Manager, who will answer questions and offer support navigating the treatment process
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Sarepta Support

Next up: Find support throughout your journey

Now that you’re familiar with the treatment process, get connected to a dedicated resource who can offer support for each step.

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Andrew 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.