Last reviewed 2026-04-20 · How we verify

Fabrazyme (AGALSIDASE BETA)

Fabrazyme works by replacing the deficient enzyme alpha-Gal A to break down and remove toxic globotriaosylceramide.

Fabrazyme (Agalsidase Beta) is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme developed by Genzyme, currently owned by the same company. It is a small molecule modality approved by the FDA in 2003 for the treatment of Fabry's disease. Fabrazyme works by replacing the deficient enzyme alpha-Gal A in patients with Fabry's disease, allowing for the breakdown and removal of globotriaosylceramide, a toxic substance that accumulates in the body. The commercial status of Fabrazyme is patented, and it is not yet available as a generic. Key safety considerations include infusion reactions, allergic reactions, and increased risk of kidney problems.

At a glance

Mechanism of action

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

Approved indications

• Fabry's disease

Boxed warnings

• WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate FABRAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue FABRAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur. [see Warnings and Precautions (5.1) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate FABRAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue FABRAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )

Common side effects

• Infusion-associated reactions
• Upper respiratory tract infection
• Chills
• Pyrexia
• Headache
• Cough
• Paresthesia
• Fatigue
• Peripheral edema
• Dizziness
• Rash
• Pain in extremity

Key clinical trials

• A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (PHASE3)
• Fabry Disease Registry & Pregnancy Sub-registry
• Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease (PHASE3)
• A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
• Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
• A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (PHASE4)
• PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion (PHASE1)
• China Post-marketing Surveillance (PMS) Study of Fabrazyme® (PHASE4)

Primary sources

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Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Fabrazyme CI brief — competitive landscape report
• Fabrazyme updates RSS · CI watch RSS
• Sanofi portfolio CI