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ELEVIDYS slows disease progression

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ELEVIDYS delivered faster time-to-rise speeds over 3 years1,2,4,25,27

ELEVIDYS 1-year results
ELEVIDYS 1-year results

ELEVIDYS-treated patients had a faster time to rise from the floor1,25

Change in TTR from floor from baseline to 1 year in Study 3 (EMBARK)
Change in TTR from floor from baseline to 1 year in Study 3 (EMBARK)
ELEVIDYS 3-year results
ELEVIDYS 3-year results

ELEVIDYS-treated patients had a 6.05-second improvement in time to rise from the floor2,4,27

Change in TTR from floor from baseline to 3 years in Study 3 (EMBARK)
Change in TTR from floor from baseline to 3 years in Study 3 (EMBARK)

* MMRM methods account for missing data in these analyses.

External control comparison was a prespecified exploratory outcome. Data are exploratory and should be interpreted with caution.

The TTR from floor (>5 seconds) is a key clinical predictor of decline24

Andrew received ELEVIDYS in a clinical trial in 2019

ELEVIDYS improved 10MWR speed over 3 years1,2,4,25,27

ELEVIDYS 1-year results
ELEVIDYS 1-year results

ELEVIDYS-treated patients demonstrated improved walk/run speed1,25

Change in time of 10MWR from baseline to 1 year in Study 3 (EMBARK)
Change in time of 10MWR from baseline to 1 year in Study 3 (EMBARK)
ELEVIDYS 3-year results
ELEVIDYS 3-year results

ELEVIDYS-treated patients showed a 2.70-second improvement in walk/run speed2,4,27

Change in time of 10MWR from baseline to 3 years in Study 3 (EMBARK)
Change in time of 10MWR from baseline to 3 years in Study 3 (EMBARK)

* MMRM methods account for missing data in these analyses.

External control comparison was a prespecified exploratory outcome. Data are exploratory and should be interpreted with caution.

The time to complete the 10MWR test (>10 seconds) is a key clinical predictor of decline21

MMRM=mixed model repeated measures; MWR=meter walk/run; TTR=time to rise.

ELEVIDYS delivered faster time-to-rise speeds over 3 years1,2,4,25,27

ELEVIDYS 1-year results
ELEVIDYS 1-year results

ELEVIDYS-treated patients had a faster time to rise from the floor1,25

Change in TTR from floor from baseline to 1 year in Study 3 (EMBARK)
Change in TTR from floor from baseline to 1 year in Study 3 (EMBARK)
ELEVIDYS 3-year results
ELEVIDYS 3-year results

ELEVIDYS-treated patients had a 6.05-second improvement in time to rise from the floor2,4,27

Change in TTR from floor from baseline to 3 years in Study 3 (EMBARK)
Change in TTR from floor from baseline to 3 years in Study 3 (EMBARK)

* MMRM methods account for missing data in these analyses.

External control comparison was a prespecified exploratory outcome. Data are exploratory and should be interpreted with caution.

The TTR from floor (>5 seconds) is a key clinical predictor of decline24

ELEVIDYS improved 10MWR speed over 3 years1,2,4,25,27

ELEVIDYS 1-year results
ELEVIDYS 1-year results

ELEVIDYS-treated patients demonstrated improved walk/run speed1,25

Change in time of 10MWR from baseline to 1 year in Study 3 (EMBARK)
Change in time of 10MWR from baseline to 1 year in Study 3 (EMBARK)
ELEVIDYS 3-year results
ELEVIDYS 3-year results

ELEVIDYS-treated patients showed a 2.70-second improvement in walk/run speed2,4,27

Change in time of 10MWR from baseline to 3 years in Study 3 (EMBARK)
Change in time of 10MWR from baseline to 3 years in Study 3 (EMBARK)

* MMRM methods account for missing data in these analyses.

External control comparison was a prespecified exploratory outcome. Data are exploratory and should be interpreted with caution.

The time to complete the 10MWR test (>10 seconds) is a key clinical predictor of decline21

MMRM=mixed model repeated measures; MWR=meter walk/run; TTR=time to rise.

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ELEVIDYS helped preserve motor function over 3 years1,2,4,25-27

ELEVIDYS 1-year results
ELEVIDYS 1-year results

ELEVIDYS-treated patients scored higher on the NSAA1,4,25*

Change in NSAA total score from baseline to 1 year in Study 3 (EMBARK)
Change in NSAA total score from baseline to 1 year in Study 3 (EMBARK)

Patients in Study 1 had similar results1,4,26

The mean change (LS) in NSAA total score from baseline to 48 weeks was 1.7 (95% CI: 0.5 to 3.0) in the ELEVIDYS group (n=19) compared with 0.9 (95% CI: -0.3 to 2.2) in the placebo group (n=21). The 0.8-point difference did not reach statistical significance (P=0.37)

ELEVIDYS 2- and 3-year results
flag-over-2+3-years-mobile

Results showed a durable slowing of disease progression2,27

Change in NSAA total score from baseline in Study 3 (EMBARK)
Change in NSAA total score from baseline in Study 3 (EMBARK)

* The NSAA provides a broad assessment of physical function using a 3-point scoring method (0=inability, 1=ability with assistance, 2=complete independence). Scoring may limit the sensitivity of NSAA to changes over short time spans.15

† MMRM methods account for missing data in these analyses.

External control comparison was a prespecified exploratory outcome. Data are exploratory and should be interpreted with caution.

A minimal clinically important difference in NSAA ranges from 2.3 to 2.9 points in ambulatory patients aged 7 to 10 years with Duchenne28

LS=least square; MMRM=mixed model repeated measures; NSAA=North Star Ambulatory Assessment.

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Totality of evidence demonstrates impact of ELEVIDYS on motor function over 3 years1,2,25

ELEVIDYS 1-year results
ELEVIDYS 1-year results

Results favored ELEVIDYS across multiple functional measures1,25

Functional outcomes from Study 3 (EMBARK) at 1 year
Functional outcomes from Study 3 (EMBARK) at 1 year

* Time to ascend n=62; 100MWR n=59.

† Time to ascend n=60; 100MWR n=57.

Carter received ELEVIDYS in 2023
ELEVIDYS 3-year results
ELEVIDYS 3-year results

ELEVIDYS slowed disease progression2

Functional outcomes from Study 3 (EMBARK) at 3 years
Change in time of 10MWR from baseline to 3 years in Study 3 (EMBARK)

‡ Results for time to ascend 4 steps and 100MWR remained stable over 2 years; there was no external control comparison available.

External control comparison was a prespecified exploratory outcome. Data are exploratory and should be interpreted with caution.

MWR=meter walk/run; NSAA=North Star Ambulatory Assessment; TTR=time to rise.

See the impact of ELEVIDYS on:

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.
  • 15. Muntoni F, Domingos J, Manzur AY, et al. Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy. PLoS One. 2019;14(9):e0221097. doi:10.1371/journal.pone.0221097
  • 21. Muntoni F, Signorovitch J, Sajeev G, et al. DMD genotypes and motor function in Duchenne muscular dystrophy: a multi-institution meta-analysis with implications for clinical trials. Neurology. 2023;100(15):e1540-e1554. doi:10.1212/WNL.0000000000201626
  • 24. Zambon AA, Gupta VA, Ridout D, et al. Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy. Dev Med Child Neurol. 2022;64(8):979-988. doi:10.1111/dmcn.15176
  • 25. Mendell JR, Muntoni F, McDonald CM, et al. AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nat Med. 2025;31(1):332-341. doi:10.1038/s41591-024-03304-z
  • 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
  • 27. Mendell JR, Muntoni F, McDonald CM, et al. Two-year outcomes following delandistrogene moxeparvovec treatment in ambulatory patients with Duchenne muscular dystrophy: Phase 3 EMBARK trial. Neurol Ther. 2026;15:545-559. doi:10.1007/s40120-025-00879-8
  • 28. Gupta VA, Pitchforth JM, Domingos J, et al. Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy. PLoS One. 2023;18(4):e0283669. doi:10.1371/journal.pone.0283669

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.