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Caring for your child after treatment

Caring for your child after treatment

Caring for your child after treatment

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

People treated with ELEVIDYS are monitored weekly for at least 3 months, or longer if needed, to support their well-being. Your doctor will order blood tests to monitor:

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

More frequent monitoring may be required.

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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 for at least 3 hours after treatment

  2. During the first two weeks
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    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. During the first two months
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    During the first two months

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

    • An immune response affecting muscles (immune-mediated myositis)
    • Inflammation of the heart (myocarditis)
    • Liver injury (there were no cases of liver failure in the clinical trials)

     

    To help reduce the risk of side effects, your child will need to take treatment-related steroids. Your child will start these shortly before the infusion day and will continue for at least 2 months.*
  4. During the first three months
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    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 – or the dose – based on the person’s response to ELEVIDYS, including helping address any changes in liver function tests. It’s important to follow your doctor’s advice on ELEVIDYS treatment-related steroids, including during the tapering period.

In clinical trials, most side effects occurred within 2 months of treatment, but it is recommended that people treated with ELEVIDYS are monitored for 3 months or longer, if needed. 

It is important to report any side effects to your doctor.

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ELEVIDYS support team
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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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Boy who received treatment with ELEVIDYS
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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).

Please see full Prescribing Information for ELEVIDYS.

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