Skip to main content
Lotties
1 - src: /sites/elevidyshcp/files/2024-08/ELE-HCP-2.1-2.2-Crecent-10-001.json / styles: top: 1050px;right: -358px;width: 583px;height: 1602px; / flip x: 0
2 - src: /sites/elevidyshcp/files/2024-08/ELE-HCP-3.1-3.3-Crescent-15blue-001_1.json / styles: top: 1400px;left: -210px;width: 403px;height: 1537px; / flip x: 0
3 - src: /sites/elevidyshcp/files/2024-09/ELE-HCP-3.0-Crecent-12-001.json / styles: top: 4841px;right: -433px;width: 667px;height: 2007px;transform: scaleX(-1); / flip x: 1
4 - src: /sites/elevidyshcp/files/2024-10/ELE-HCP-2.1-Crescent-9update-001.json / styles: top: -90px;left: -142px;width: 492px;height: 759px; / flip x: 0

Meaningful treatment benefit in ambulatory patients1

Statistically significant and highly expressed ELEVIDYS micro-dystrophin in treated patients1

Clinical trials confirmed targeted ELEVIDYS micro-dystrophin expression in skeletal muscle cells across ambulation status and ages studied.
 

ELEVIDYS micro-dystrophin expression shown in three studies
ELEVIDYS micro-dystrophin expression shown in three studies

* All patients received 1.33 x 1014 vg/kg, as measured by ddPCR.  

† Change from baseline was statistically significant.  

‡ Adjusted for muscle content. Control was level of wild-type (normal) dystrophin in normal muscle.    

ddPCR=droplet digital polymerase chain reaction.

Patients with Duchenne typically have little to no endogenous dystrophin.3

A clear association between Week 12 micro-dystrophin expression and clinical outcome assessment (assessed by change from baseline on the Performance of Upper Limb version 2) in non-ambulatory patients has not been established.1

Assessment of ELEVIDYS micro-dystrophin levels can be meaningfully influenced by differences in sample processing, analytical technique, reference materials, and quantitation methodologies. Therefore, valid comparisons of ELEVIDYS micro-dystrophin measurements obtained from different assays cannot be made.1
 

Boy who received treatment with ELEVIDYS

Impact on motor function

— AMBULATORY PATIENTS —

Patients treated with ELEVIDYS scored higher on the NSAA, compared with placebo. While results favored ELEVIDYS, they did not reach statistical significance.1
 

Change in NSAA total score from baseline to 48 weeks
Change in NSAA total score from baseline to 48 weeks
Change in NSAA total score from baseline to 52 weeks
Change in NSAA total score from baseline to 52 weeks

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.12

LS=least squares; NSAA=North Star Ambulatory Assessment.
 

Patients treated with ELEVIDYS demonstrated a clinically relevant change in the ability to rise from the floor and walk/run compared with placebo2,25
 

Change in total time to rise from floor from baseline to 52 weeks
Change in time of 10MWR from baseline to 52 weeks
Change in total time to rise from floor from baseline to 52 weeks
Change in time of 10MWR from baseline to 52 weeks

Both the TTR from floor (>5 seconds) and time to complete the 10MWR test (>10 seconds) are key clinical predictors of decline.20,21

MWR=meter walk/run; TTR=time to rise.

Totality of evidence confirms a clinically relevant treatment benefit1
 

time function test chart
time function test chart

Results favored ELEVIDYS across critical measures of motor function.

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

 

see results in non-ambulatory patients

References:

  • 1. ELEVIDYS. Prescribing information. Sarepta Therapeutics, Inc; August 2024.
  • 2. Data on file. Sarepta Therapeutics, Inc. Cambridge, MA.
  • 3. Asher DR, Thapa K, Dharia SD, et al. Clinical development on the frontier: gene therapy for Duchenne muscular dystrophy. Expert Opin Biol Ther. 2020;20(3):263-274. doi:10.1080/14712598.2020.1725469
  • 12. 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
  • 20. 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
  • 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
  • 25. Mendell JR, Muntoni F, McDonald CM, et al. AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nat Med. 2024. 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

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.

Read More Close