A NOVEL GENE THERAPY
FOR DUCHENNE

ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle observed in patients treated with ELEVIDYS. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

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Helpful resources

HCP treatment guide HCP treatment guide
Patient treatment guide Patient treatment guide
Patient treatment guide – Spanish Patient treatment guide – Spanish

Educational videos

Learn how Elevidys works Learn how ELEVIDYS works
How To Prepare Dose And Infuse See how to prepare, dose, and infuse ELEVIDYS

Dedicated support for your patients

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Our SareptAssist patient support program helps your patients explore eligibility, coverage, financial assistance options, and treatment logistics and includes dedicated patient offerings specific to gene therapy to facilitate treatment. Get your patients started in just 3 steps:

More information about ELEVIDYS is coming soon. In the meantime, if you have questions about ELEVIDYS or how to take the next steps for your patients, contact our team at 1-888-SAREPTA ( 1-888-727-3782). Case Managers are available Monday through Friday, 8:30am - 6:30pm ET.

SareptAssist is a resource available only to those who have been prescribed a Sarepta product; enrollment in the program is required to receive this assistance. Participation in SareptAssist is not necessary to receive treatment with ELEVIDYS. Program is only available in the US.

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ELEVIDYS is an infusion, which is only administered at select treatment centers throughout the country. Use the tool below to locate treatment centers. Please note that this information is regularly updated and may change.

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Treatment centers that administer ELEVIDYS are independently owned and operated. Sarepta Therapeutics does not have oversight of the medical care provided by these centers.

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Important Safety Information

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

WARNINGS AND PRECAUTIONS:
Acute Serious Liver Injury:

Indication

ELEVIDYS is indicated for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval based on the expression of ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS. 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:
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 the 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.
  • 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 subjects 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 function tests increased, 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 for ELEVIDYS.