Onasemnogene Abeparvovec
Onasemnogene abeparvovec is a gene therapy designed to treat spinal muscular atrophy (SMA) by delivering a functional copy of the SMN1 gene to patients who have mutations in this gene. It is administered as a single intravenous infusion and is primarily used in pediatric patients with SMA.
A one-time gene therapy for spinal muscular atrophy that delivers a functional SMN gene via AAV9 vector.
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
- M09AX09
- RxNorm
- 2170226
- Generic
- Onasemnogene Abeparvovec
- Class
- atc-m09
- Brands
- Zolgensma, Itvisma
- 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
- 2.0 × 10^14 vector genomes (maximum dose)
- Maximum
- consult physician
- Frequency
- single one-time infusion
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 1.1 × 10^14 vector genomes per kilogram
- Maximum
- consult physician
- Frequency
- single one-time infusion
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2.0 × 10^14 vector genomes (maximum dose)
- Maximum
- consult physician
- Frequency
- single one-time infusion
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
7 groupspregnancy
generalavoid if possible
avoid use during pregnancy unless benefits significantly outweigh risks; counsel patients on potential fetal risks
regular fetal monitoring including ultrasound assessments
lactation
generalnot recommended
breastfeeding should be discontinued prior to treatment and avoided for a period after administration
no specific monitoring established
pediatric_age
infantsweight-based dosing required
indicated for pediatric patients under 2 years with bi-allelic mutations in SMN1 gene; administer as single IV infusion; pre-treatment corticosteroids required
liver function tests baseline and regular monitoring for at least 3 months; platelet counts; cardiac monitoring
geriatric
elderlynot established
not typically indicated; geriatric patients not studied in clinical trials; use only if benefits outweigh risks
comprehensive geriatric assessment; liver and cardiac monitoring
renal_impairment
mild to moderateno specific adjustment recommended
use with caution; renal function should be assessed prior to treatment
monitor renal function tests periodically post-administration
Use with caution and monitor for adverse effects.
hepatic_impairment
mildclose monitoring required
use with caution; patients with pre-existing hepatic impairment may be at increased risk for hepatotoxicity
frequent liver function tests including AST, ALT, bilirubin; monitor for signs of hepatic injury
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate to severegenerally contraindicated
not recommended in patients with moderate to severe hepatic impairment due to significantly increased risk of hepatotoxicity
enhanced liver function monitoring if use cannot be avoided
Use of this medication in this population requires careful consideration of risks and benefits.
Statistics and clinical data
Drug class
Onasemnogene abeparvovec is an adeno-associated virus (AAV) vector-based gene therapy
Common uses
Indicated for treatment of pediatric patients with SMA
Administration
One-time gene therapy treatment
FDA approval year
Initial FDA approval date
Onasemnogene abeparvovec is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric patients with bi-allelic mutations in the SMN1 gene resulting in insufficient SMN protein expression.
This recombinant AAV9-based gene therapy delivers a functional copy of the human SMN gene, enabling cells to produce survival motor neuron (SMN) protein, which is deficient in SMA patients due to SMN1 mutations.
Administered as a single intravenous infusion. Liver function monitoring and platelet count assessment are important components of treatment monitoring. Patients should be evaluated for pre-existing AAV9 antibodies prior to treatment.
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