• Miscellaneous musculoskeletal disorders

Elevidys Generic Name & Formulations

General Description

Delandistrogene moxeparvovec-rokl 1.33x10^13 vg/mL; susp for IV infusion; preservative-free.

Pharmacological Class

Adeno-associated virus vector-based gene therapy.

How Supplied

Customized kit—1 (10–70 vials + alcohol wipes)


Generic Availability


Mechanism of Action

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 ssDNA 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 Elevidys micro-dystrophin protein. 

Elevidys Indications


In ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. 

Elevidys Dosage and Administration


Not applicable.


≤3yrs or ≥6yrs: not established. Select patients with anti-AAVrh74 total binding antibody titers <1:400. Give as a single-dose IV infusion using a syringe infusion pump over approx. 1–2 hours (or longer based on discretion). 4–5yrs: 1.33×1014vg/kg. Infusion rate: <10mL/kg/hr. Initiate a corticosteroid regimen starting 1 day prior to Elevidys infusion: see full labeling.

Elevidys Contraindications


Deletion in exon 8 and/or exon 9 in the DMD gene.

Elevidys Boxed Warnings

Not Applicable

Elevidys Warnings/Precautions


Risk of acute serious liver injury (esp. preexisting liver impairment, chronic hepatic condition, or acute hepatic viral infection). Monitor liver function (eg, clinical exam, GGT, total bilirubin) prior to infusion, weekly for the 1st 3 months, then as clinically indicated until unremarkable results. Obtain platelets and troponin-I prior to infusion, weekly for the 1st 2 weeks (platelets) and weekly for the 1st month (troponin-I), then as clinically indicated. Increased risk of serious systemic immune response; delay infusion in those with concurrent infections until resolved. Risk of immune-mediated myositis (esp. deletions in the DMD gene in exons 1–17 and/or exons 59–71); if symptoms occur, consider additional immunomodulatory treatment (eg, calcineurin-inhibitor). Perform baseline anti-AAVrh74 antibody testing (Total Binding Antibody ELISA) prior to infusion. Elevated anti-AAVrh74 total binding antibody titers ≥1:400: not recommended. Consult a specialist if acute serious liver injury/liver failure is suspected or troponin elevations accompanied by signs/symptoms. Hepatic impairment or elevated GGT. Elderly. Pregnancy, nursing mothers: not applicable.

Elevidys Pharmacokinetics

See Literature

Elevidys Interactions


Complete all immunizations at least 4 weeks prior to initiating corticosteroid regimen.

Elevidys Adverse Reactions

Adverse Reactions

Vomiting, nausea, increased LFT, pyrexia, thrombocytopenia; acute serious liver injury, immune-mediated myositis, myocarditis.

Elevidys Clinical Trials

See Literature

Elevidys Note

Not Applicable

Elevidys Patient Counseling

See Literature