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agalsidase beta (GZ419828)
Agalsidase beta is a recombinant human alpha-galactosidase A enzyme that breaks down globotriaosylceramide (Gb3) accumulation in cells.
Agalsidase beta is a recombinant human alpha-galactosidase A enzyme that breaks down globotriaosylceramide (Gb3) accumulation in cells. Used for Fabry disease.
At a glance
| Generic name | agalsidase beta (GZ419828) |
|---|---|
| Also known as | Fabrazyme, Fabrazyme® |
| Sponsor | Sanofi |
| Drug class | Enzyme replacement therapy |
| Target | Alpha-galactosidase A |
| Modality | Small molecule |
| Therapeutic area | Rare genetic disease / Lysosomal storage disorder |
| Phase | FDA-approved |
Mechanism of action
Fabry disease is caused by deficiency of the enzyme alpha-galactosidase A, leading to accumulation of the glycosphingolipid Gb3 in various tissues. Agalsidase beta is a recombinant replacement enzyme that catalyzes the hydrolysis of Gb3, reducing pathological substrate accumulation in endothelial cells, cardiomyocytes, and renal cells. This enzyme replacement therapy helps prevent or slow progressive organ damage characteristic of Fabry disease.
Approved indications
- Fabry disease
Common side effects
- Infusion-related reactions
- Fever
- Chills
- Headache
- Nausea
- Hypersensitivity reactions
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)
- A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (PHASE4)
- China Post-marketing Surveillance (PMS) Study of Fabrazyme® (PHASE4)
- To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease (PHASE4)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- agalsidase beta (GZ419828) CI brief — competitive landscape report
- agalsidase beta (GZ419828) updates RSS · CI watch RSS
- Sanofi portfolio CI