Alglucosidase Alfa
Alglucosidase alfa is an enzyme replacement therapy used to treat Pompe disease (glycogen storage disease type II), a rare genetic disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). The medication is administered via intravenous infusion and helps reduce glycogen accumulation in muscles and tissues, improving respiratory function and motor outcomes in patients with both infantile-onset and late-onset forms of the disease.
Alglucosidase alfa is an enzyme replacement therapy for Pompe disease that replaces deficient GAA enzyme to break down harmful glycogen buildup in cells.
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
- A16AB07
- RxNorm
- 629565
- Generic
- Alglucosidase Alfa
- Class
- atc-a16
- Brands
- Lumizyme
- 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
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
10 groupspregnancy
first trimesterno specific adjustment
use only if clearly needed and benefits outweigh potential risks
regular prenatal monitoring
Use with caution and monitor for adverse effects.
pregnancy
second trimesterno specific adjustment
continue therapy if needed for maternal disease management
regular fetal growth monitoring
Use with caution and monitor for adverse effects.
pregnancy
third trimesterno specific adjustment
continue as needed and discuss delivery plans with healthcare team
fetal monitoring and assessment for preterm labor
Use with caution and monitor for adverse effects.
lactation
postpartumno adjustment recommended
decide whether to discontinue breastfeeding or discontinue drug considering importance to mother
monitor infant for adverse effects
pediatric_age
infantsweight-based dosing at 20 mg/kg weekly for infantile-onset
initiate treatment as soon as diagnosis confirmed
regular cardiac and respiratory monitoring, growth assessment
pediatric_age
childrenweight-based dosing at 20 mg/kg weekly
standard dosing for late-onset Pompe disease in pediatric patients
monitor respiratory function, motor development
geriatric
elderly 65 74no specific adjustment
use standard dosing with close monitoring of cardiac and respiratory function
regular cardiac and pulmonary assessment
Use with caution and monitor for adverse effects.
renal_impairment
mild renalno dose adjustment required
standard dosing recommended
standard monitoring
renal_impairment
moderate renalno specific adjustment recommended
use with caution and monitor renal function
Use with caution and monitor for adverse effects.
hepatic_impairment
mild hepaticno specific adjustment
use with caution as enzyme is metabolized systemically
monitor liver function tests
Use with caution and monitor for adverse effects.
Statistics and clinical data
Drug class
Pharmacological classification of Alglucosidase Alfa
Common uses
Therapeutic indications for Alglucosidase Alfa
FDA approval year
FDA approval date for Lumizyme/Alglucosidase Alfa
Mechanism of action
Pharmacodynamic mechanism of Alglucosidase Alfa
Treatment of Pompe disease (glycogen storage disease type II, acid maltase deficiency) in patients of all ages, including infantile-onset and late-onset forms of the disease.
Alglucosidase alfa is a recombinant human acid alpha-glucosidase (rhGAA) that provides an exogenous source of the deficient lysosomal enzyme. After intravenous administration, the enzyme is taken up by cells and transported to lysosomes, where it hydrolyzes glycogen into glucose, thereby reducing glycogen accumulation in cardiac, skeletal, and respiratory muscles.
Administered as intravenous infusions, typically every 2 weeks. Hypersensitivity reactions including anaphylaxis may occur during or after infusion. Patients should be monitored closely during infusions and pre-medication may be considered. Immune responses to the enzyme may develop, potentially affecting treatment efficacy.
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