Agalsidase Beta

FABRAZYME (agalsidase beta) provides an exogenous source of α-galactosidase A in Fabry disease patients. Agalsidase beta is internalized and transported into lysosomes where it exerts enzymatic activity and reduces accumulated GL-3.

• FABRAZYME ® is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. FABRAZYME is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease.

• None. None.contraindicated

Administration of FABRAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. ( 2.1 ) The recommended dosage is 1 mg/kg body weight given every two weeks as an intravenous infusion. ( 2.2 ) See the full prescribing information for rechallenge, preparation, storage, and administration instructions. ( 2.3 , 2.4 , 2.5 , 2.6 ) 2.1 Recommendations Prior to FABRAZYME Treatment Administration of FABRAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis [see Warnings and Precautions (5.1) ]. Initiate FABRAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment [see Warnings and Precautions (5.1) ]. Prior to FABRAZYME administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids [see Warnings and Precautions (5.1 , 5.2) ] . FABRAZYME must be reconstituted and diluted prior to use [see Dosage and Administration (2.4) ] 2.2 Recommended Dosage and Administration The recommended dosage of FABRAZYME is 1 mg/kg body weight infused every two weeks as an intravenous infusion. The initial recommended infusion rate is 0.25 mg/min (15 mg/hour) [see Dosage and Administration (2.6) ] . 2.3 Rechallenge Instructions Patients who have had a positive skin test to FABRAZYME or who have tested positive for anti-FABRAZYME IgE may be successfully rechallenged with FABRAZYME.

Infusion-Associated Reactions : If a severe infusion-associated reaction occurs, discontinue FABRAZYME immediately and initiate appropriate medical treatment. ( 5.2 ) 5.1 Hypersensitivity Reactions Including Anaphylaxis Life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in FABRAZYME-treated patients. In clinical trials and postmarketing safety experience with FABRAZYME, approximately 1% of patients developed anaphylaxis or severe hypersensitivity reactions. Reactions have included localized angioedema (including swelling of the face, mouth, and throat), bronchospasm, hypotension, generalized urticaria, dysphagia, rash, dyspnea, flushing, chest discomfort, pruritus, and nasal congestion. In clinical trials with FABRAZYME, 10 of 238 patients developed IgE antibodies or skin test reactivity specific to FABRAZYME. Two of six patients in the rechallenge study discontinued treatment with FABRAZYME prematurely due to recurrent infusion-associated reactions. Four serious infusion-associated reactions occurred in three patients during FABRAZYME infusions, including bronchospasm, urticaria, hypotension, and development of FABRAZYME-specific antibodies. Other infusion-associated reactions occurring in more than one patient during the study included rigors, hypertension, nausea, vomiting, and pruritus.

The following clinically significant adverse reactions are described elsewhere in labeling: Hypersensitivity Reactions Including Anaphylaxis [see Warnings and Precautions (5.1) ] Infusion-Associated Reactions [see Warnings and Precautions (5.2) ] Most common adverse reactions (≥20%) are: upper respiratory tract infection, chills, pyrexia, headache, cough, paresthesia, fatigue, peripheral edema, dizziness, and rash. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genzyme at 1-800-633-1610 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in clinical trials of a drug cannot be directly compared to rates in the clinical trial of another drug and may not reflect the rates observed in patients in clinical practice. The data described below reflect exposure of 80 patients, ages 16 to 61 years, to 1 mg/kg FABRAZYME every two weeks in two separate double-blind, placebo-controlled clinical trials, for periods ranging from 1 to 35 months (mean 15.5 months). All 58 patients enrolled in one of the two studies continued into an open-label extension study of FABRAZYME treatment for up to 54 additional months. Patients were treated with antipyretics and antihistamines prior to the infusions. Most Common Adverse Reactions Table 2 enumerates adverse reactions that occurred during the double-blind treatment periods of the two placebo-controlled trials (Study 1 and Study 2) [see Clinical Studies (14) ] .

8.1 Pregnancy Risk Summary Available data from a pregnancy sub-study within the Fabry Disease registry, post-marketing case reports, and case series with FABRAZYME use during pregnancy have not identified a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes (see Data ). Reproduction studies performed in rats at doses up to 68 times the human dose have revealed no evidence of effects on embryo-fetal development (see Data ) . The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Human Data Available data from a pregnancy sub-study within the Fabry Disease registry, post-marketing case reports, and case series with FABRAZYME use during pregnancy have not identified a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes. In the Fabry Disease registry pregnancy sub-study, 33 pregnancies exposed to FABRAZYME prior to or during pregnancy had a known outcome; 5 were reported as exposed in the first trimester.

Metabolism

The pharmacokinetics of FABRAZYME in clinical studies with adult and pediatric patients with Fabry disease is summarized in Table 3. FABRAZYME exhibited nonlinear pharmacokinetics following intravenous infusions at 0.3 (30% of the approved recommended dosage), 1 mg/kg, and 3 mg/kg (3 times the approved recommended dosage) in adult patients.

• Apr 24, 2003BLABLA103979Genzyme

1. 1Pyrexia5175.8%
2. 2Pain4985.5%
3. 3Malaise4825.4%
4. 4Chills4745.3%
5. 5Nausea4595.1%
6. 6Fatigue4194.7%
7. 7Dyspnoea4094.6%
8. 8Headache3684.1%
9. 9Vomiting3654.1%
10. 10Weight Decreased3283.7%
11. 11Pain In Extremity3073.4%
12. 12Infusion Related Reaction2973.3%
13. 13Dizziness2713.0%
14. 14Weight Increased2622.9%
15. 15Condition Aggravated2562.9%

The 10 most recently updated of 38 ClinicalTrials.gov registrations naming Agalsidase Beta as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
  Active not recruiting · Observational · 78 enrolled · Sanofi
  NCT06052800updated 2026-05-15
• Fabry Disease Registry & Pregnancy Sub-registry
  Recruiting · Observational · 9,000 enrolled · Genzyme, a Sanofi Company
  NCT00196742updated 2026-04-28
• A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
  Active not recruiting · Phase 3 · Interventional · 104 enrolled · Sanofi
  NCT05280548updated 2026-03-10
• Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
  Completed · Phase 3 · Interventional · 20 enrolled · Bio Sidus SA
  NCT05843916updated 2026-02-18
• A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
  Completed · Observational · 82 enrolled · Amicus Therapeutics
  NCT04281537updated 2026-02-05
• Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
  Enrolling by invitation · Observational · 100 enrolled · NPO Petrovax
  NCT06880250updated 2025-12-31
• A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
  Completed · Phase 4 · Interventional · 8 enrolled · Sanofi
  NCT06019728updated 2025-10-21
• PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion
  Completed · Phase 1 · Interventional · 24 enrolled · Bio Sidus SA
  NCT05343715updated 2025-10-15
• China Post-marketing Surveillance (PMS) Study of Fabrazyme®
  Completed · Phase 4 · Interventional · 22 enrolled · Genzyme, a Sanofi Company
  NCT05054387updated 2025-09-11
• Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
  Recruiting · Phase 3 · Interventional · 24 enrolled · ISU Abxis Co., Ltd.
  NCT06081062updated 2024-07-29

Frequently asked questions

How does Agalsidase Beta work?

FABRAZYME (agalsidase beta) provides an exogenous source of α-galactosidase A in Fabry disease patients. Agalsidase beta is internalized and transported into lysosomes where it exerts enzymatic activity and reduces accumulated GL-3.

What is Agalsidase Beta used for?

According to FDA labeling, Agalsidase Beta carries indications including: FABRAZYME ® is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. FABRAZYME is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Agalsidase Beta?

Agalsidase Beta is classified as Enzymes, Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme, Mechanism of Action, Lipid Metabolism Alteration.

What are the brand names for Agalsidase Beta?

Agalsidase Beta is marketed under brand names including Fabrazyme.

What are the contraindications for Agalsidase Beta?

Agalsidase Beta labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.