Avalglucosidase Alfa
Avalglucosidase Alfa is a recombinant human acid alpha-glucosidase enzyme replacement therapy designed to treat Pompe disease, a rare genetic disorder characterized by the accumulation of glycogen in cellular lysosomes due to enzyme deficiency. This medication is used in both pediatric and adult patients to help break down glycogen and prevent its harmful buildup in muscles and tissues.
Avalglucosidase Alfa is an enzyme replacement therapy for Pompe disease that helps restore glycogen breakdown in affected patients.
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
- A16AB22
- RxNorm
- 2565814
- Generic
- Avalglucosidase Alfa
- Class
- atc-a16
- Brands
- Nexviazyme
- 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
- 20 mg/kg every 2 weeks
- Maximum
- consult physician
- Frequency
- every 2 weeks
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 20 mg/kg every 2 weeks
- Maximum
- consult physician
- Frequency
- every 2 weeks
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 20 mg/kg every 2 weeks
- Maximum
- consult physician
- Frequency
- every 2 weeks
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
9 groupspregnancy
first trimesteravoid if possible
use only if benefits outweigh potential risks to fetus
regular fetal monitoring recommended
Use with caution and monitor for adverse effects.
pregnancy
second trimesteravoid if possible
continue therapy only if benefits outweigh risks
regular fetal monitoring and uterine activity assessment
Use with caution and monitor for adverse effects.
pregnancy
third trimesteravoid if possible
use only if benefits outweigh potential risks
regular fetal monitoring and assessment for preterm labor
Use with caution and monitor for adverse effects.
pediatric_age
pediatricapproved for use in pediatric patients; dosing based on body weight
regular monitoring of cardiac and respiratory function
geriatric
elderlyno specific adjustment
use with caution due to potential comorbidities
monitor cardiac and respiratory function closely
Use with caution and monitor for adverse effects.
renal_impairment
mild to moderateno adjustment needed
no dose adjustment required for mild to moderate renal impairment
standard monitoring
renal_impairment
severeno specific adjustment
use with caution, limited data in severe renal impairment
close monitoring recommended
Use with caution and monitor for adverse effects.
hepatic_impairment
mild to moderateno specific adjustment
use with caution, limited data available
monitor liver function tests
Use with caution and monitor for adverse effects.
lactation
breastfeedingdiscontinue or not initiate
not recommended during breastfeeding due to unknown excretion in breast milk
monitor infant for adverse effects
Statistics and clinical data
Drug class
Classification as enzyme replacement therapy for Pompe disease
Common uses
Therapeutic indication for enzyme replacement therapy
FDA approval
FDA approval date for Avalglucosidase Alfa (Nexviazyme)
Mechanism of action
Enzyme replacement mechanism in glycogen metabolism
Long-term treatment of Pompe disease (glycogen storage disease type II, acid maltase deficiency, glycogenosis type II) in pediatric and adult patients with confirmed diagnosis
Provides exogenous acid alpha-glucosidase enzyme that is internalized by cells and transported to lysosomes, where it catalyzes the breakdown of glycogen into glucose, reducing glycogen accumulation in affected tissues
Administered via intravenous infusion; patients should be monitored for infusion-associated reactions and hypersensitivity responses; regular assessment of clinical response and tolerability is recommended; dosage individualized based on patient factors
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