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Required monitoring

Weekly safety monitoring is required for at least 3 months (or longer, if clinically indicated) to assess liver function, platelet count, and troponin-I levels.1

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Safety considerations1

Adverse reactions typically seen within the first 2 weeks following infusion include nausea, vomiting, thrombocytopenia, and pyrexia. Vomiting may occur as early as on the day of the infusion.

Adverse reactions occurring within the first 2 months include immune-mediated myositis and liver injury.

Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during and for at least 3 hours after the infusion, and slow/stop the infusion to administer treatment, as needed.

See Important Safety Information for additional safety considerations when infusing ELEVIDYS.

Postinfusion assessments1

Liver function monitoring

Liver function

  • Acute serious liver injury has been observed with ELEVIDYS. Administration of ELEVIDYS may result in elevations of liver enzymes (eg, GGT, ALT) and total bilirubin, typically seen within 8 weeks
  • Monitor liver function weekly for the first 3 months via clinical exam and analysis of GGT and total bilirubin levels. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam and GGT and total bilirubin levels return to near baseline levels)
Platelet count monitoring

Platelet count

  • Transient, mild, asymptomatic decrease in platelet count has been observed following ELEVIDYS administration
  • Monitor platelet counts weekly for the first 2 weeks
Myocarditis monitoring

Myocarditis

  • Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials
  • If a patient experiences myocarditis, those with preexisting left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Patients with moderate to severe LVEF impairment have not been studied in clinical trials with ELEVIDYS
  • Monitor troponin-I weekly for the first month. More frequent monitoring may be required in the presence of cardiac symptoms, such as chest pain or shortness of breath

Continue monitoring after the recommended schedule if clinically indicated.


ALT=alanine transaminase; GGT=gamma-glutamyl transferase.

 

Continuing the required corticosteroid regimen1

Immune responses to the AAVrh74 vector can occur after administration of ELEVIDYS. To reduce the risk associated with an immune response, patients must continue treatment with systemic corticosteroids for at least 60 days after the infusion, unless earlier tapering is clinically indicated.

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Recommended corticosteroid regimen dose modification for liver function abnormalities following ELEVIDYS infusion*

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* GGT≥150 U/L and/or other clinically significant liver function abnormalities (eg, total bilirubin >2 × ULN) following infusion. For GGT or bilirubin elevations that do not respond to these oral corticosteroid increases, IV bolus corticosteroids may be considered. 

† Corticosteroids other than prednisone and prednisolone have not been studied for use as a peri-ELEVIDYS infusion corticosteroid.

It is critical that patients adhere to a pre- and postinfusion corticosteroid regimen to reduce the risk of an adverse immune response.1

Boy who received treatment with ELEVIDYS

Postinfusion guidance for caregivers1

Counsel patients and caregivers on what to expect postinfusion and reiterate the importance of compliance with the required corticosteroids and monitoring schedule.

  1. Speak with your patients or caregivers about the risk of infusion-related reactions, 
    acute serious liver injury, immune-mediated myositis, and myocarditis

    Advise patients and caregivers to contact you immediately if:

    • The patient experiences a fast heart rate, fast breathing, swollen lips, being short of breath, nostrils widening, hives, red and blotchy skin, itchy or inflamed lips, rash, vomiting, nausea, chills, or fever, as these may be signs of an infusion-related reaction
    • 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
    • The patient experiences any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, difficulty breathing, or difficulty speaking, as these may be symptoms of myositis
    • The patient begins to experience chest pain and/or shortness of breath, as this may be a sign of myocarditis

  2. Remind patients and caregivers about the importance of corticosteroid treatment to help reduce the risk of an immune response to the ELEVIDYS vector (AAVrh74)

  3. Reinforce the importance of the weekly monitoring of liver function, troponin-I levels, and platelet counts

  4. Advise patients and caregivers that an infection (eg, cold, flu, gastroenteritis, otitis media, bronchiolitis, etc) before or after infusion could lead to more serious complications. Remind them to contact a healthcare provider immediately if symptoms suggestive of infection are observed (eg, coughing, wheezing, sneezing, runny nose, sore throat, or fever)

  5. Remind patients, caregivers, and family members about proper hand hygiene when they come into direct contact with the patient’s body waste. Note that they should place potentially contaminated materials in a sealable bag and dispose of it in the regular trash. Encourage this behavior for at least 1 month after infusion to avoid vector shedding

References:

  • 1. ELEVIDYS. Prescribing information. Sarepta Therapeutics, Inc; August 2024.

Indication

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age:

  • For patients who are ambulatory and have a confirmed mutation in the DMD gene
  • For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.

The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

 

Important Safety Information

CONTRAINDICATION:
ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

WARNINGS AND PRECAUTIONS:
Infusion-related Reactions:

  • Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate.
  • ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available.
  • Discontinue infusion for anaphylaxis. 

Acute Serious Liver Injury:

  • Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks.
  • Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (eg, acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.
  • Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels).
  • Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended.

Immune-mediated Myositis:

  • In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed.
  • Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction.
  • Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [eg, calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur.

Myocarditis:

  • Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials.
  • If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath.
  • Advise patients to contact a physician immediately if they experience cardiac symptoms.

Preexisting Immunity against AAVrh74:

  • In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies.
  • Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.
  • ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400.

Adverse Reactions:

  • The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.

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.

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