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Safety evaluated in >200 trial patients with real-world experience in >1,000, some >5 years postinfusion1,4,30*

Clinical trial safety data were gathered from 200 participants, including 156 males who received ELEVIDYS and 62 who received placebo.1 More than 1,000 people have received ELEVIDYS worldwide,4* and ongoing trials and postmarketing experience continue to evaluate the efficacy and safety of ELEVIDYS.2,26

*Includes worldwide clinical trial and US commercial use through November 2025.

The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increase.1

Adverse reactions occurring in ELEVIDYS-treated subjects and at least twice as frequently than with placebo (Study 3 [EMBARK], 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%

Most adverse events occurred within 90 days of infusion, with no new safety signals observed between 1 and 3 years1,2,26

† 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.

Timing for when adverse reactions were typically seen1

Time periodAdverse reactions
Within 2 weeks of infusionNausea, vomiting (as early as on infusion day), thrombocytopenia, pyrexia
Within 1 month of infusionMyocarditis, increased troponin-I levels
Within 2 months of infusionImmune-mediated myositis, liver injury

Post-marketing experience1

The following adverse reactions have been identified during post-approval use of ELEVIDYS. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.

  • Hepatobiliary disorders: acute liver injury, acute liver failure, including fatal outcome and life-threatening mesenteric vein thrombosis.
  • Infections and infestations: bacterial and viral respiratory infections, including fatal outcome
  • Immune system disorders: infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration
  • Musculoskeletal and connective tissue disorders: immune-mediated myositis
Provide caregivers with the ELEVIDYS Medication Guide, inform them of the Important Safety Information, and urge them to contact a doctor immediately if an adverse event occurs. See postinfusion monitoring for more caregiver counseling information

Cardiac outcomes

ELEVIDYS 3-year results
ELEVIDYS 3-year results

Prespecified endpoint: Echocardiogram LVEF over 3 years in Part 1 of Study 3 (EMBARK) in participants treated at ages 4 to <8 years; ELEVIDYS2¶

 

N

Baseline

1 year

2 years

3 years

Echocardiogram LVEF (%), mean (SD)

50

65.3 (5.7)

65.2 (5.0)

64.5 (6.9)

60.5 (3.6)

 

N

Baseline

Echocardiogram LVEF (%), mean (SD)

50

65.3 (5.7)

 

1 years

2 years

3 years

Echocardiogram LVEF (%), mean (SD)

65.2 (5.0)

64.5 (6.9)

60.5 (3.6)

A normal LVEF is generally between 50% and 70%.31 In boys with Duchenne, the mean age at which LVEF falls below 55% is approximately 15 years, though it can be earlier.32 

Data are exploratory and should be interpreted with caution.

¶ No external control comparison available. 
ALT=alanine transaminase; AST=aspartate transferase; GGT=gamma-glutamyl transferase; GLDH=glutamate dehydrogenase; LVEF=left ventricular ejection fraction.

References:

  • 1. ELEVIDYS. Prescribing information. Sarepta Therapeutics, Inc; November 2025.

  • 2. Mendell JR, Muntoni F, McDonald CM, et al. Delandistrogene moxeparvovec in Duchenne muscular dystrophy: functional and safety outcomes up to 3 years post-infusion in the EMBARK study. Poster presented at: Muscular Dystrophy Association Clinical & Scientific Conference; March 8-11, 2026; Orlando, FL.

  • 4. Data on file. Sarepta Therapeutics, Inc. Cambridge, MA.

  • 26. Mendell JR, Shieh PB, McDonald CM, et al. Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy. Front Cell Dev Biol. 2023;11:1167762. doi:10.3389/fcell.2023.1167762

  • 30. Mendell JR, Sahenk Z, Lehman KJ, et al. Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: a phase1/2a nonrandomized trial. Muscle Nerve. 2024;69(1):93-98. doi:10.1002/mus.27955

  • 31. Cleveland Clinic. Ejection fraction. Updated April 14, 2026. Accessed May 8, 2026. https://my.clevelandclinic.org/health/articles/16950-ejection-fraction

  • 32. James KA, Gralla J, Ridall LA, et al. Left ventricular dysfunction in Duchenne muscular dystrophy. Cardiol Young. 2020; 30(2):171-176. doi:10.1017/S1047951119002610

Indication

ELEVIDYS is indicated for the treatment of ambulatory patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.

Limitations of Use
ELEVIDYS is not recommended in patients with:

  • Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limit of normal or total bilirubin > the upper limit of normal not due to Gilbert's syndrome) or active hepatic viral infection due to the high risk of acute serious liver injury and acute liver failure.
  • Recent vaccination (within 4 weeks of treatment) due to immunogenicity and potential safety concerns.
  • Active or recent (within 4 weeks) infections due to safety concerns.

Please see full Prescribing Information, including Boxed Warning, and Medication Guide.

Important Safety Information, including Boxed Warning

BOXED WARNING: Acute Serious Liver Injury and Acute Liver Failure

Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred.
Patients with preexisting liver impairment may be at higher risk. 

Prior to infusion, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroids before and after ELEVIDYS infusion. Continue to monitor liver function weekly for the first 3 months after infusion and continue until results are unremarkable. 

Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion.

Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected.

CONTRAINDICATION
ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene. 

WARNINGS AND PRECAUTIONS
Acute Serious Liver Injury and Acute Liver Failure
See Boxed Warning.

  • Acute serious liver injury marked by elevations of liver enzymes (e.g., GGT, ALT) and total bilirubin and acute liver failure has occurred with ELEVIDYS. Onset of the liver injury typically begins within 8 weeks of ELEVIDYS administration. In non-ambulatory patients treated with ELEVIDYS, acute liver failure with fatal outcome has occurred in the clinical and post-marketing settings.
  • Life-threatening mesenteric vein thrombosis, complicated by bowel ischemia and necrosis, and portal hypertension have been reported following acute liver injury associated with ELEVIDYS in a non-ambulatory patient.
  • Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury or acute liver failure. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.
  • Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated.

Serious infections

  • Increased susceptibility to serious infections may occur due to concomitant administration of corticosteroid regimen and additional immunosuppressants, and ELEVIDYS. Serious respiratory infections, including with fatal outcomes, have occurred in patients taking immunosuppressant corticosteroids required for ELEVIDYS administration.
  • Monitor patients for signs and symptoms of infection before and after ELEVIDYS administration and treat appropriately.
  • Administer immunizations according to best clinical practices and immunization guidelines prior to initiation of the corticosteroid regimen required before ELEVIDYS infusion.
  • Avoid administration of ELEVIDYS to patients with active infections.

Myocarditis

  • Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed within 24 hours to more than 1 year following ELEVIDYS infusion.
  • 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, until results return to near baseline levels or stabilize.
  • 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.

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 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. 

Immune-mediated Myositis

  • Immune-mediated myositis, including serious and life-threatening events, has occurred approximately 1 month following ELEVIDYS infusion. Signs and symptoms include severe muscle weakness, including dysphagia, dyspnea, dysphonia, and hypophonia.
  • Severe to life-threatening immune-mediated myositis has been reported in patients with deletions including portions of exons 1-17 and/or exons 59-71 of the DMD gene.
  • Regardless of genetic mutation, advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, dysphonia, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment based on patient's clinical presentation and medical history if these symptoms occur.

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 the 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 ≥1:400.

ADVERSE REACTIONS

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

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.