Delandistrogene Moxeparvovec

ELEVIDYS is the recombinant gene therapy product that is comprised of a non-replicating, recombinant, adeno-associated virus (AAV) serotype rh74 (AAVrh74) capsid and a single-stranded DNA expression cassette flanked by inverted terminal repeats (ITRs) derived from AAV2. The cassette contains: 1) an MHCK7 gene regulatory component comprising a creatine kinase 7 promoter and an α-myosin heavy chain enhancer, and 2) the DNA transgene encoding the engineered micro-dystrophin protein.

• ELEVIDYS is indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD), who are ambulatory and have a confirmed mutation in the DMD gene [see Clinical Pharmacology ( 12.2 ), Clinical Studies ( 14 )] . ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who are ambulatory and have a confirmed mutation in the DMD gene.

• 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 [see Warnings and Precautions ( 5.5 )] . 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.contraindicated

For single-dose intravenous infusion only. ELEVIDYS is for single-dose intravenous infusion only. Select patients for treatment with ELEVIDYS with anti-AAVrh74 total binding antibody titers <1:400. ( 2.1 ) Postpone in patients with active or recent (within 4 weeks) infections. ( 2.1 ) Assess liver function, platelet counts and troponin-I before ELEVIDYS infusion. ( 2.1 ) Recommended dosage: 10 to 70 kg: 1.33 × 10 14 vector genomes (vg) per kg of body weight; 70 kg or greater: 9.31 × 10 15 vg. ( 2.2 ) One day prior to infusion, initiate a corticosteroid regimen for a minimum of 60 days. Recommend modifying corticosteroid dose for patients with liver function abnormalities. ( 2.2 ) Administer as an intravenous infusion over 1-2 hours. Infuse at a rate of less than 10 mL/kg/hour. ( 2.4 ) 2.1 Critical Dosing Information Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion. Prior to ELEVIDYS infusion: Select patients for treatment with ELEVIDYS with anti-AAVrh74 total binding antibody titers <1:400. An FDA-authorized test for the detection of anti-AAVrh74 total binding antibodies is not currently available. Currently available tests may vary in accuracy and design. Avoid ELEVIDYS administration in patients with elevated anti-AAVrh74 total binding antibody titers (≥1:400) [Clinical Pharmacology ( 12.6 )] .

Serious Infections: Serious infections with fatal outcomes may occur due to concomitant administration of corticosteroids, additional immunosuppressants, and ELEVIDYS. Monitor patients for signs and symptoms of infection; treat appropriately. ( 5.2 ) Myocarditis: Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed. Monitor troponin-I before ELEVIDYS infusion, and weekly for the first month after ELEVIDYS infusion. ( 5.3 ) Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during administration and for at least 3 hours after end of infusion. If symptoms occur, slow or stop the infusion and give appropriate treatment. Once symptoms resolve, restart infusion at a slower infusion rate. Discontinue infusion for anaphylaxis. ( 2.4 , 5.4 ) Immune-mediated Myositis: Severe to life-threatening immune-mediated myositis has been reported with ELEVIDYS in patients with deletions including portions of exons 1 to 17 and /or exons 59 to 71 of the DMD gene. Consider additional immunomodulatory treatment if symptoms of myositis occur (e.g., unexplained increased muscle pain, tenderness, or weakness). ( 5.5 ) Pre-existing Immunity against AAVrh74: Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ( 5.6 ) 5.1 Acute Serious Liver Injury and Acute Liver Failure 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.

Most common adverse reactions (incidence ≥5%) were vomiting and nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc., at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety data described in this section reflect exposure to a one-time intravenous infusion of ELEVIDYS in 156 male patients with a confirmed mutation of the DMD gene in four clinical studies, including one completed open-label study, one ongoing open-label study, and two studies that included a double-blind, placebo-controlled period. Prior to ELEVIDYS infusion, patients in the ELEVIDYS treatment group had a mean age of 6.7 years (range: 3 to 20) and mean weight of 24.6 kg (range: 12.5 to 80.1). 144 patients received the recommended dose of 1.33 × 10 14 vg/kg, and 12 received a lower dose. Table 3 below presents adverse reactions from these four clinical studies. The most common adverse reactions (incidence ≥5%) across all studies are summarized in Table 3 .

8.1 Pregnancy Risk Summary ELEVIDYS is not intended for use in pregnant women. In the U.S. general population, the estimated background risks of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. 8.2 Lactation Risk Summary There is no information available on the presence of ELEVIDYS in human milk, the effects on the breastfed infant, or the effects on milk production. 8.4 Pediatric Use The safety and effectiveness of ELEVIDYS for the treatment of Duchenne muscular dystrophy has been established in pediatric patients at least 4 years of age with a confirmed mutation in the DMD gene. The use of ELEVIDYS in pediatric patients was supported by evidence from three adequate and well controlled clinical studies which included 144 pediatric patients aged 4 years of age and older [see Adverse Reactions ( 6 ), Clinical Pharmacology ( 12.2 ), Clinical Studies ( 14 )] . 8.5 Geriatric Use The safety and efficacy of ELEVIDYS in geriatric patients with DMD have not been studied. 8.6 Hepatic Impairment The safety and efficacy of ELEVIDYS in patients with hepatic impairment or elevated GGT have not been studied. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.

Metabolism

Vector Distribution and Vector Shedding Nonclinical Data Biodistribution of ELEVIDYS was evaluated in tissue samples collected from healthy mice and DMD mdx mice following intravenous administration in toxicology studies. At 12 weeks following ELEVIDYS administration at dose levels of 1.33 ×10 14 to 4.02 ×10 14 vg/kg, vector DNA was detected in all major organs with the highest quantities detected in the liver, followed by lower levels in the heart, adrenal glands, skeletal muscle, and aorta.

1. 1Vomiting5017%
2. 2Gamma-glutamyltransferase Increased4215%
3. 3Nausea3613%
4. 4Alanine Aminotransferase Increased3211%
5. 5Aspartate Aminotransferase Increased2910%
6. 6Blood Creatine Phosphokinase Increased269.0%
7. 7Hepatic Enzyme Increased269.0%
8. 8Liver Injury238.0%
9. 9Troponin I Increased217.3%
10. 10Gait Disturbance175.9%
11. 11Pyrexia165.6%
12. 12Fatigue155.2%
13. 13Myalgia144.9%
14. 14Asthenia134.5%
15. 15Fall124.2%

The 10 most recently updated of 11 ClinicalTrials.gov registrations naming Delandistrogene Moxeparvovec as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
  Not yet recruiting · Phase 4 · Interventional · 20 enrolled · Sarepta Therapeutics, Inc.
  NCT07542314updated 2026-05-26
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
  Active not recruiting · Phase 3 · Interventional · 148 enrolled · Sarepta Therapeutics, Inc.
  NCT05881408updated 2026-05-22
• A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
  Active not recruiting · Phase 2 · Interventional · 13 enrolled · Hoffmann-La Roche
  NCT06128564updated 2026-05-11
• An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
  Enrolling by invitation · Observational · 500 enrolled · Sarepta Therapeutics, Inc.
  NCT06270719updated 2026-05-04
• A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
  Recruiting · Phase 1 · Interventional · 83 enrolled · Sarepta Therapeutics, Inc.
  NCT04626674updated 2026-04-06
• A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
  Enrolling by invitation · Phase 3 · Interventional · 400 enrolled · Sarepta Therapeutics, Inc.
  NCT05967351updated 2026-02-06
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
  Terminated · Phase 1 · Interventional · 5 enrolled · Sarepta Therapeutics, Inc.
  NCT06241950updated 2025-11-20
• A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
  Terminated · Phase 1 · Interventional · 3 enrolled · Sarepta Therapeutics, Inc.
  NCT06597656updated 2025-09-04
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
  Completed · Phase 3 · Interventional · 126 enrolled · Sarepta Therapeutics, Inc.
  NCT05096221updated 2025-07-08
• A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
  Completed · Phase 1 · Phase 2 · Interventional · 41 enrolled · Sarepta Therapeutics, Inc.
  NCT03769116updated 2024-11-14

Frequently asked questions

How does Delandistrogene Moxeparvovec work?

ELEVIDYS is the recombinant gene therapy product that is comprised of a non-replicating, recombinant, adeno-associated virus (AAV) serotype rh74 (AAVrh74) capsid and a single-stranded DNA expression cassette flanked by inverted terminal repeats (ITRs) derived from AAV2. The cassette contains: 1) an MHCK7 gene regulatory component comprising a creatine kinase 7 promoter and an α-myosin heavy chain enhancer, and 2) the DNA transgene encoding the engineered micro-dystrophin protein.

What is Delandistrogene Moxeparvovec used for?

According to FDA labeling, Delandistrogene Moxeparvovec carries indications including: ELEVIDYS is indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD), who are ambulatory and have a confirmed mutation in the DMD gene [see Clinical Pharmacology ( 12.2 ), Clinical Studies ( 14 )] . ELEVIDYS is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who are ambulatory and have a confirmed mutation in the DMD gene.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Delandistrogene Moxeparvovec?

Delandistrogene Moxeparvovec is classified as Other drugs for disorders of the musculo-skeletal system, Gene Alterations, Gene Editing or Modification, Unknown Physiological Effect.

What are the brand names for Delandistrogene Moxeparvovec?

Delandistrogene Moxeparvovec is marketed under brand names including Elevidys.

What are the contraindications for Delandistrogene Moxeparvovec?

Delandistrogene Moxeparvovec labeling lists contraindications including: 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 [see Warnings and Precautions ( 5.5 )] . 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.. Always consult the full prescribing information and a clinician.