Agalsidase Beta (Monograph)

Brand name: Fabrazyme
Drug class: Enzymes

Medically reviewed by Drugs.com on Aug 10, 2025. Written by ASHP.

Introduction

Biosynthetic (recombinant DNA origin) form of human α-galactosidase A.^{1 2 3 4 5 6 7 8 9}

Uses for Agalsidase Beta

Fabry Disease

Treatment of adults and pediatric patients ≥2 years of age with Fabry disease (designated an orphan drug by FDA for this use).^{1 2 3 4 5 6 7 8 9 12 13 14}

Management recommendations for pediatric patients and adults with Fabry disease include consideration of early enzyme replacement therapy (i.e., agalsidase beta), use of adjunctive therapies to manage complications, ongoing monitoring for evidence of organ involvement and patient response, and involvement of an experienced multidisciplinary team.^{10 11}

Agalsidase Beta Dosage and Administration

General

Premedication and Prophylaxis

• Consider premedication with antihistamines, antipyretics, and/or corticosteroids.¹

Dispensing and Administration Precautions

• Administer agalsidase beta under the supervision of a clinician knowledgeable in the management of hypersensitivity reactions including anaphylaxis.¹

• Administer agalsidase beta in a healthcare setting with appropriate medical monitoring and support measures readily available.¹

Administration

IV Administration

Administer by IV infusion following reconstitution and dilution.¹

Administer through a low-protein-binding 0.2-µm filter. ¹

Do not infuse in the same IV line with other products. ¹

Reconstitution

Determine appropriate number of vials for reconstitution via the actual body weight of the patient and recommended dose.¹

Remove vials from refrigeration and allow to sit at room temperature (20–25°C) for approximately 30 minutes prior to use. ¹

Reconstitute vial containing 35 or 5 mg of agalsidase beta powder with 7.2 or 1.1 mL, respectively, of Sterile Water for Injection to provide a solution containing 5 mg of agalsidase beta per mL.¹

Direct the diluent slowly toward the side of the vial; gently roll and tilt vial.¹ Do not shake or agitate.¹

Reconstituted solution should be clear and colorless.¹ Discard if particulate matter is visible or solution discolored.¹

Dilution

Remove the volume of diluent equal to the total required volume of reconstituted agalsidase beta solution from an infusion bag of 0.9% sodium chloride.

Slowly add reconstituted agalsidase beta solution to the bag to a total volume based on patient weight. (See Table 1.) Final concentration of diluted solution is 0.2 to 0.7 mg/mL of agalsidase beta.¹

Gently invert infusion bag to mix solution; do not agitate or shake.¹

Rate of Administration

Infuse at an initial rate of 0.25 mg/minute (15 mg/hr).¹

Can increase infusion rate in increments of 0.05 to 0.08 mg/minute (3 to 5 mg/hour) with each subsequent infusion in patients weighing ≥30 kg in the absence of hypersensitivity and/or infusion-associated reactions.¹

For patients weighing <30 kg, maximum infusion rate is 0.25 mg/minute (15 mg/hour).¹

Dosage

Pediatric Patients

Fabry’s Disease

IV

Pediatric patients ≥2 years of age: 1 mg/kg every 2 weeks as an IV infusion.¹ Initial recommended infusion rate is 0.25 mg/minute (15 mg/hour).¹

Adults

Fabry’s Disease

IV

1 mg/kg every 2 weeks as an IV infusion.¹ Initial recommended infusion rate is 0.25 mg/minute (15 mg/hour).¹

Rechallenge Instructions

If a patient has a positive skin test to agalsidase beta or tested positive for anti-agalsidase beta IgE, may rechallenge the patient.¹ Initial rechallenge should involve a low agalsidase beta dose at a reduced infusion rate (e.g., 0.5 mg/kg at 0.01 mg/minute [6 mg/hour]).¹ Once infusion tolerated, dose may be increased to approved dose of 1 mg/kg and infusion rate increased by slow upward titration (i.e., doubling every 30 minutes to a maximum infusion rate of 0.25 mg/minute) as tolerated.¹

Special Populations

Manufacturer states no special dosage recommendations for geriatric patients or those with hepatic or renal impairment.¹

Cautions for Agalsidase Beta

Contraindications

• None.¹

Warnings/Precautions

Warnings

Hypersensitivity Reactions Including Anaphylaxis

Life-threatening hypersensitivity reactions, including anaphylaxis, reported (see Boxed Warning).¹ Hypersensitivity reactions occurred more frequently in adults with persistent anti-drug antibodies and those with high antibody titer as compared to adults who were antibody-negative.¹

Anaphylaxis with enzyme replacement reported both early on and after extended duration of therapy.¹ An experienced clinician in the management of hypersensitivity reactions, including anaphylaxis, should supervise initiation of agalsidase beta therapy in a healthcare setting with appropriate medical monitoring and support measures.¹ Consider pretreatment with antihistamines, antipyretics, and/or corticosteroids.¹

Discontinue agalsidase beta and initiate appropriate medical treatment immediately in patients who experience a severe hypersensitivity reaction.¹ Consider risks and benefits of readministration following occurrence of a severe hypersensitivity reaction.¹ Inform patients of the symptoms of hypersensitivity reactions and to seek immediate medical care if these symptoms occur.¹

In patients who experience a serious hypersensitivity reaction during therapy, consider testing for IgE antibodies as well as the benefits and risks of continued therapy in patients with confirmed anti-drug antibodies.¹ Tests for antibodies against agalsidase beta are not currently marketed; if testing is necessary, contact Genzyme at 1-800-745-4447.¹ Patients who have tested positive for agalsidase beta-specific IgE antibodies or who have a positive skin test to agalsidase beta may be rechallenged.¹ Rechallenge should only occur under the direct supervision of qualified personnel with appropriate medical monitoring and support measures readily available.¹

Other Warnings/Precautions

Infusion-associated Reactions

Infusion-associated reactions, some severe, may occur.¹

Consider pretreatment with antihistamines, antipyretics, and/or corticosteroids to reduce risk of infusion-associated reactions.¹ Despite pretreatment, reactions may still occur.¹ Infusion-associated reactions tend to decline in frequency with continued administration; however, reactions may occur despite an extended duration of therapy.¹ Appropriate medical monitoring and support measures should be readily available during administration.¹

Discontinue agalsidase beta immediately and initiate appropriate medical treatment if a severe infusion-associated reaction occurs.¹ After considering risks and benefits, readministration of agalsidase beta may occur after a severe infusion-associated reaction; however, monitor patients closely.¹ Following a mild or moderate infusion-associated reaction, consider temporarily holding infusion, slowing infusion rate, and/or reducing agalsidase beta dosage.¹

Compromised cardiac function may occur in patients with advanced Fabry disease, which may predispose these patients to an increased risk of severe complications from infusion-associated reactions.¹ Monitor patients closely during administration.¹

Immunogenicity

In clinical studies of patients with classic Fabry disease, patients developed antibodies to agalsidase beta.¹ In addition, neutralizing antibodies that inhibited in vitro agalsidase beta catalytic activity and cellular uptake occurred.¹ Majority of patients with antibodies developed them within initial 3 months of therapy; antibody titers generally declined over time.¹ Female patients generally had a reduced incidence of antibodies and lower antibody titers as compared to male patients.¹

Specific Populations

Pregnancy

Clinical data from a Fabry Disease registry pregnancy sub-study, postmarketing case reports, and case series involving administration of agalsidase beta during pregnancy did not reveal a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes.¹

Lactation

Agalsidase beta detected in human milk in small amounts; however, data insufficient to determine effects on a breastfed infant or milk production.¹ The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for the drug and any potential adverse effects of the drug on the breastfed child or from the underlying maternal condition.¹

Pediatric Use

Safety and efficacy established in pediatric patients based on results from adequate and well-controlled studies in adults, a single-arm, open-label study in 16 pediatric patients (8 to 16 years of age) with Fabry disease, and additional data in 24 pediatric patients (2 to 7 years of age) with the condition.¹ Overall safety profile similar between adults and pediatric patients.¹

Geriatric Use

Insufficient experience in patients ≥65 years of age to determine whether geriatric patients respond differently than younger adults.¹

Common Adverse Effects

Most common adverse reactions (≥20%) include upper respiratory tract infection, chills, pyrexia, headache, cough, paresthesia, fatigue, peripheral edema, dizziness, rash.¹

Drug Interactions

No formal drug interaction studies performed to date.¹

Some clinicians state that biosynthetic forms of α-galactosidase should not be administered concurrently with chloroquine, amiodarone, monobenzone, or gentamicin because of a theoretical risk of inhibited intracellular α-galactosidase activity.⁵

Agalsidase Beta Pharmacokinetics

Absorption

Exhibits nonlinear pharmacokinetics following IV infusion of various weight-based doses in adults.¹ AUC and maximum plasma concentration increased greater than dose proportional with increasing doses.¹

Stability

Storage

Parenteral

Powder for Injection

2–8°C.¹

Following reconstitution and dilution, 2–8°C up to 24 hours; however, immediate use following reconstitution and dilution preferred since product contains no preservatives.¹

Actions

• Provides exogenous source of α-galactosidase that catalyzes the hydrolysis of glycosphingolipids, including GL-3, in patients with Fabry’s disease. ^{2 3 5}

• Agalsidase beta is internalized and transported into lysosomes where it exerts enzymatic activity and reduces accumulated GL-3.¹

Advice to Patients

• Advise patients that life-threatening hypersensitivity, including anaphylaxis, and infusion-associated reactions may occur with treatment.¹ Inform patients of the signs and symptoms of these reactions and to immediately seek medical care if these occur.¹

• Stress importance of informing clinicians of existing or contemplated concomitant therapy, including prescription and OTC drugs and dietary supplements (e.g., herbal products).¹

• Advise patients to inform clinicians if they are or plan to become pregnant or to breast-feed.¹ Inform pregnant or lactating patients exposed to agalsidase beta to report the exposure by calling 1-800-633-1610, option 1.¹

• Inform patients that a registry has been established to monitor the variability and progression of Fabry’s disease and to evaluate long-term treatment effects of agalsidase beta.¹ Patients may contact the registry by visiting [Web] or by calling 1-800-745-4447, extension 15500.¹

• Inform patients of other important precautionary information.¹

Additional Information

The American Society of Health-System Pharmacists, Inc. represents that the information provided in the accompanying monograph was formulated with a reasonable standard of care, and in conformity with professional standards in the field. Readers are advised that decisions regarding use of drugs are complex medical decisions requiring the independent, informed decision of an appropriate health care professional, and that the information contained in the monograph is provided for informational purposes only. The manufacturer’s labeling should be consulted for more detailed information. The American Society of Health-System Pharmacists, Inc. does not endorse or recommend the use of any drug. The information contained in the monograph is not a substitute for medical care.

Preparations

Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.

Related/similar drugs

Biological Products Related to agalsidase beta

Find detailed information on biosimilars for this medication.

Medical Disclaimer