BLAINTRAVENOUSVIALPriority Review
Approved
Apr 2003
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
19
Data completeness
6/8 — Strong
Priority Review
FABRAZYME (agalsidase beta) by Sanofi is (agalsidase beta) provides an exogenous source of α-galactosidase a in fabry disease patients. Approved for fabry disease. First approved in 2003.
Drug data last refreshed 18h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
Fabrazyme (agalsidase beta) is an intravenous enzyme replacement therapy approved in 2003 for the treatment of Fabry disease, a rare lysosomal storage disorder. The drug provides an exogenous source of α-galactosidase A enzyme that is internalized into lysosomes where it reduces accumulated globotriaosylceramide (GL-3), the pathologic substrate in Fabry disease. Fabrazyme represents the standard-of-care enzyme replacement approach for this ultra-rare genetic disorder affecting predominantly male patients and some female carriers.
$0.0BPart D
Mechanism of Action
(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.
Pharmacologic Class:
Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme
Indications (1)
Clinical Trials (19)
A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
Started Nov 2023
8 enrolled
Fabry's Disease
Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
Started Sep 2023
78 enrolled
Fabry Disease
Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
Started Apr 2023
24 enrolled
Fabry Disease
Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
Started Dec 2022
20 enrolled
Fabry Disease
China Post-marketing Surveillance (PMS) Study of Fabrazyme®
Started Oct 2021
22 enrolled
Fabry Disease
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.