FABRAZYME (agalsidase beta)
FABRAZYME is indicated for the treatment of Fabry Disease.
How FABRAZYME Works
Fabrazyme provides a supplemental source of α-galactosidase A to patients who are deficient in this enzyme. The medication is internalized by cells and transported into the lysosomes, which are the cell's digestive components. Once inside the lysosomes, the drug exerts enzymatic activity to reduce the accumulation of globotriaosylceramide (GL-3).
Development Insights
Details
- Status
- Prescription
- First Approved
- 2003-04-24
- Patent Cliff
- 2028
- Revenue
- $278M (Q4-2025)
- Routes
- INTRAVENOUS
- Dosage Forms
- VIAL
FABRAZYME Approval History
What FABRAZYME Treats
1 indicationsFABRAZYME is approved for 1 conditions since its original approval in 2003. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Fabry Disease
FABRAZYME Boxed 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 react...
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 )
FABRAZYME Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to FABRAZYME
FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
5 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05054387 | LPS16583 U1111-1255-4881 | Ph 4 | completed | China Post-marketing Surveillance (PMS) Study of Fabrazyme® |
| NCT02795676 BALANCE results posted | PB-102-F20 | Ph 3 | completed | Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function |
| NCT01196871 results posted | AT1001-013 | Ph 2 | completed | Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease |
| NCT00701415 FIELD results posted | AGAL06207 2007-005668-28, EFC12821 | Ph 3 | completed | A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms |
| NCT01650779 results posted | AGAL19412 | Ph 4 | completed | A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
FABRAZYME FDA Label Details
Indications & Usage
FDA Label (PDF)FABRAZYME is indicated for the treatment of Fabry Disease.
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...
Pro Intelligence Preview
Deep insights for FABRAZYME
Revenue Insights
- • Q4-2025: $278M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2028
- • Generic/biosimilar risk
Trial Analysis
- • 6 total trials
- • Stage: Declining
Competitive Landscape
- • 2 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment