Section 16 · Monograph
Delandistrogene moxeparvovec
Reviewed Nov 2026FDA label
ElevidysAAVrh74 gene therapy
- Forms
- IV: single-dose weight-based kit
- Dose
- One-time weight-based IV infusion; BOXED WARNING acute liver injury/failure; ambulatory patients age 4+ only; contraindicated with DMD exon 8 and/or 9 deletions
- MOA
- Delivers a micro-dystrophin gene
Black Box Warning
- Serious liver injury and acute liver failure, including fatal outcomes, have been reported — monitor liver function closely after infusion
Indications
- Treatment of Duchenne muscular dystrophy in ambulatory patients 4 years of age and older with a confirmed DMD gene mutation (non-ambulatory indication removed in the November 2025 labeling revision)FDA-labeled
Mechanism of Action
- AAVrh74 vector delivering a micro-dystrophin gene to muscle to produce a shortened functional dystrophin protein
Full Dosing
Adult / usual
- One-time IV infusion with weight-based vector-genome dosing per label
- Systemic corticosteroid regimen before and after infusion per label
Hepatic
- Not recommended in patients with preexisting liver impairment, recent vaccination, or recent or active infection
Contraindications
- Deletion involving DMD exon 8 and/or exon 9 (risk of immune-mediated myositis)
Drug Interactions
- Immunosuppression from corticosteroids increases infection risk
- Live vaccines should be timed per label
Adverse Effects
- Acute serious liver injury and liver failure
- Vomiting, nausea, fever, fatigue
- Myocarditis and troponin elevation
- Immune-mediated myositis
- Infusion reactions and complement activation
Monitoring Parameters
- Weekly liver function tests for at least 3 months after infusion
- Weekly troponin-I for about 1 month
- Remain near an appropriate medical facility for at least 2 months after infusion
Pregnancy & Lactation
- Not applicable to the pediatric indication; limited data