Drug updated on 9/4/2024
Dosage Form | Injection (intravenous; 5 mg, 35mg) |
Drug Class | Hydrolytic lysosomal enzymes |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease.
Latest News
Summary
- Fabrazyme (agalsidase beta) is indicated for the treatment of adult and pediatric patients aged 2 years and older with confirmed Fabry disease.
- This summary is based on the review of two systematic review(s)/meta-analysis(es). [1-2]
- ERT with agalsidase beta significantly decreases globotriaosylceramide levels in various biological compartments, including plasma, urine, and specific kidney, heart, and skin cell types.
- ERT with agalsidase beta slows the decline in estimated glomerular filtration rate (eGFR), suggesting a potential protective effect on renal function, and improves cardiac metrics by reducing or stabilizing left ventricular mass and cardiac wall thickness.
- In adult male patients, particularly those who begin treatment early, ERT with agalsidase beta shows significant clinical benefits, including improvements in nervous system outcomes, pain management, gastrointestinal symptoms, and overall quality of life.
- There is no safety information available in the reviewed studies.
- There is no population type or subgroup information available in the reviewed studies.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Fabrazyme (agalsidase beta) Prescribing Information. | 2023 | Genzyme Corporation, Cambridge, MA |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
Why systematic literature reviews in Fabry disease should include all published evidence. | 2019 | European Journal of Medical Genetics |
The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: a systematic literature review by a European panel of experts. | 2019 | Molecular Genetics and Metabolism Reports |
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
An expert consensus on practical clinical recommendations and guidance for patients with classic Fabry disease. | 2022 | Molecular Genetics and Metabolism |