Delandistrogene Moxeparvovec
Delandistrogene Moxeparvovec (ELEVIDYS) is a gene therapy treatment for Duchenne muscular dystrophy (DMD) designed to address the underlying genetic cause of the disease. It delivers a functional copy of the dystrophin gene to muscle cells using an adeno-associated virus (AAVrh74) as a delivery vehicle, allowing patients' cells to produce a shortened but functional version of the dystrophin protein.
ELEVIDYS is an AAV-based gene therapy that delivers a micro-dystrophin gene to muscle cells for treating Duchenne muscular dystrophy.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- M09AX15
- RxNorm
- 2641675
- Generic
- Delandistrogene Moxeparvovec
- Class
- atc-m09
- Brands
- Elevidys
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 2×10^14 vg/kg
- Maximum
- consult physician
- Frequency
- single dose
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2×10^14 vg/kg
- Maximum
- consult physician
- Frequency
- single dose
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2×10^14 vg/kg
- Maximum
- consult physician
- Frequency
- single dose
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
10 groupspregnancy
first trimesteravoid if possible
avoid use during pregnancy unless benefits significantly outweigh risks; discuss with patient the potential risks to the fetus
regular fetal monitoring and ultrasound assessments
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second trimesteravoid if possible
avoid use during pregnancy; consider alternative therapies
regular fetal monitoring
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
third trimesteravoid if possible
avoid use; postpone treatment until after delivery if possible
regular fetal monitoring and contraction monitoring
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingavoid
do not administer during breastfeeding; breast milk excretion unknown
monitor infant if exposure occurred
Use of this medication in this population requires careful consideration of risks and benefits.
pediatric_age
children 4 5 yearsone-time infusion at 2×10^14 vg/kg
indicated for ambulatory pediatric patients aged 4 years and older with confirmed DMD mutation; pre-infusion immune response monitoring required
baseline and ongoing liver function tests, cardiac monitoring, and anti-AAV rh74 antibody testing
pediatric_age
children under 4not established
not recommended for patients under 4 years of age; limited safety and efficacy data
requires careful risk-benefit assessment if used off-label
Use with caution and monitor for adverse effects.
geriatric
elderlynot established
not typically indicated as DMD is a pediatric-onset condition; use only if benefits outweigh risks
comprehensive cardiac and hepatic assessment if treated
renal_impairment
mild to moderateno specific dose adjustment recommended based on current data
use with caution; monitor renal function before and after infusion
baseline and periodic renal function monitoring (creatinine, BUN, eGFR)
Use with caution and monitor for adverse effects.
hepatic_impairment
milduse with caution
AAV vectors primarily transduce liver; pre-existing hepatic dysfunction may affect safety and efficacy
frequent liver function tests (ALT, AST, bilirubin); monitor for transaminitis
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate to severenot recommended
avoid use in moderate to severe hepatic impairment due to altered vector clearance and increased hepatotoxicity risk
liver function must be assessed; contraindicated if significant hepatic impairment present
Use of this medication in this population requires careful consideration of risks and benefits.
Statistics and clinical data
FDA approval year
FDA approval date for Delandistrogene Moxeparvovec (Elevidys) for treatment of Duchenne muscular dystrophy
Therapeutic class
Classification as AAV-based gene therapy for muscular dystrophy
Indication
Primary therapeutic indication for patients with confirmed DMD mutation
Target patient age
Approved age range for treatment with Elevidys
Delandistrogene Moxeparvovec is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the dystrophin gene who are ambulatory and meet other specific clinical criteria.
The therapy uses a non-replicating AAV serotype rh74 capsid to deliver a single-stranded DNA expression cassette containing a truncated dystrophin gene (micro-dystrophin) into muscle cells. Once delivered, the cells can produce a shortened but functional dystrophin protein, which helps protect muscle fibers from damage.
As a one-time intravenous infusion, immune responses to the AAV capsid must be monitored, and liver function should be assessed. Use is contraindicated in patients with certain genetic mutations and significant资肺 involvement. Treatment should be administered by experienced specialists in a qualified healthcare setting.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy