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Consistent safety results across more than 150 clinical trial patients1

Safety was evaluated in more than 200 clinical trial participants, including 156 males with a confirmed mutation in the DMD gene who received ELEVIDYS and 62 who received placebo. Limited safety data is available for non-ambulatory patients.1

Adverse reactions (incidence ≥5%) following treatment with ELEVIDYS in clinical trials1

Adverse reactions

ELEVIDYS

N=156

Vomiting

65%

Nausea

43%

Liver injury*

40%

Pyrexia

28%

Thrombocytopenia†‡

8%

In clinical trials, adverse events were medically managed with appropriate monitoring or treatment. See Postinfusion monitoring. 


* Includes: AST increased, ALT increased, GGT increased, GLDH increased, GLDH level abnormal, hepatotoxicity, hepatic enzyme increased, hypertransaminasemia, liver function test increased, liver injury, transaminases increased, blood bilirubin increased. 

† Includes: thrombocytopenia, platelet count decreased. 

‡ Transient, mild, asymptomatic decrease in platelet counts.

Adverse reactions occurring in ELEVIDYS-treated subjects and at least twice more frequently than with placebo (Study 3, Part 1)1

Adverse reactions

ELEVIDYS

n=63

Placebo

n=62

Vomiting

64%

19%

Nausea

40%

13%

Liver injury*

41%

8%

Pyrexia

32%

24%

Thrombocytopenia†‡

3%

0%

* Includes: AST increased, ALT increased, GGT increased, GLDH increased, GLDH level abnormal, hepatotoxicity, hepatic enzyme increased, hypertransaminasemia, liver function test increased, liver injury, transaminases increased. 

† Includes: platelet count decreased, thrombocytopenia. 

‡ Transient, mild, asymptomatic decrease in platelet counts.

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.1

Adverse reactions occurring within the first 2 months include immune-mediated myositis and liver injury.1

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.1

Follow the comprehensive safety protocols that were designed to help manage the occurrence of serious adverse reactions.1
Inform patients or caregivers of the Important Safety Information for ELEVIDYS and when they should contact a healthcare provider immediately if an adverse event occurs, including signs or symptoms of infusion-related reactions, acute liver injury, immune-mediated myositis, myocarditis, or symptoms of infection.1

AST=aspartate transferase; ALT=alanine transaminase; GGT=gamma-glutamyl transferase; GLDH=glutamate dehydrogenase.

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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