Agalsidase Beta
Agalsidase Beta is an enzyme replacement therapy used to treat Fabry disease, a rare genetic disorder caused by deficiency of the α-galactosidase A enzyme. This medication works by providing an exogenous source of the enzyme, which is taken up by cells and transported to lysosomes where it breaks down accumulated globotriaosylceramide (GL-3), reducing the harmful buildup that causes disease symptoms.
Agalsidase Beta is an enzyme replacement therapy for Fabry disease that reduces accumulated GL-3 by providing the missing α-galactosidase A enzyme.
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
- A16AB04
- RxNorm
- 338817
- Generic
- Agalsidase Beta
- Class
- atc-a16
- Brands
- Fabrazyme
- 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
- 1 mg/kg every 2 weeks
- Maximum
- consult physician
- Frequency
- every 2 weeks
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 1 mg/kg every 2 weeks
- Maximum
- consult physician
- Frequency
- every 2 weeks
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 1 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
11 groupspregnancy
first trimesterno specific adjustment
use only if clearly needed after benefit-risk assessment
regular prenatal care and fetal monitoring
Use with caution and monitor for adverse effects.
pregnancy
second trimesterno specific adjustment
continue therapy if benefits outweigh potential risks
routine prenatal monitoring
Use with caution and monitor for adverse effects.
pregnancy
third trimesterno specific adjustment
may continue treatment per physician assessment
fetal wellbeing monitoring
Use with caution and monitor for adverse effects.
lactation
breastfeedingunknown
discontinue breastfeeding or pump and discard milk during treatment
monitor infant for any adverse effects
Use with caution and monitor for adverse effects.
pediatric_age
children 2 121 mg/kg every 2 weeks
approved for patients 2 years and older
regular assessment of growth and development
pediatric_age
adolescents 13 171 mg/kg every 2 weeks
same dosing as adults
standard pediatric monitoring
geriatric
elderly over 65no specific adjustment
no dose adjustment required but monitor closely
monitor renal and cardiac function
Use with caution and monitor for adverse effects.
renal_impairment
mild to moderateno dose adjustment needed
standard dosing applies
standard monitoring
renal_impairment
severeno specific adjustment
use with caution and monitor closely
close monitoring of renal function
Use with caution and monitor for adverse effects.
hepatic_impairment
mildno specific data
use with caution
monitor liver function tests
hepatic_impairment
moderate to severenot studied
use only if benefits outweigh risks
close monitoring of hepatic function
Statistics and clinical data
FDA approval year
FDA approval for Fabry disease treatment
Drug class
Therapeutic classification
Common uses
Therapeutic indication for Fabry disease
Mechanism of action
Enzyme replacement therapy for glycolipid metabolism
Agalsidase Beta is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (Fabry's disease), a rare X-linked lysosomal storage disorder caused by deficiency of the α-galactosidase A enzyme.
Agalsidase Beta provides an exogenous source of α-galactosidase A enzyme. After intravenous administration, the enzyme is internalized by cells and targeted to lysosomes, where it exerts its enzymatic activity to hydrolyze accumulated globotriaosylceramide (GL-3) into galactosylceramide, thereby reducing substrate storage in various tissues.
Agalsidase Beta is administered via intravenous infusion, typically every 2 weeks. Common adverse reactions include infusion-associated reactions such as fever, chills, and flushing. Antibody formation against the enzyme may occur in some patients. Treatment should be initiated and supervised by physicians experienced in managing Fabry disease. Regular monitoring of clinical response and GL-3 levels may be performed to assess 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