Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 5 mg, 35mg) | 
| Drug Class | Hydrolytic lysozomal neutral glycosphingolipid-specific 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
- This summary is based on the review of two systematic review(s)/meta-analysis(es). [1-2]
 - Agalsidase beta as an enzyme replacement therapy (ERT) for Fabry disease facilitates cellular globotriaosylceramide (Gb3) clearance, contributing to reducing the disease burden associated with Fabry disease symptoms.
 - The presence of neutralizing anti-drug antibodies (ADAs) in ERT-treated males may reduce the effectiveness of agalsidase beta, potentially leading to disease progression in this subgroup.
 - Comparative data between agalsidase beta and agalsidase alpha indicate both therapies aid in Gb3 clearance; however, specific comparative effectiveness outcomes were not detailed in the studies.
 - Agalsidase beta therapy is associated with infusion-associated reactions, though specific details of these reactions are not provided.
 - The formation of neutralizing anti-drug antibodies (ADAs) in males treated with enzyme replacement therapy for Fabry disease is noted as a safety concern, as it may reduce the therapy's effectiveness and potentially lead to disease progression.
 
Product Monograph / Prescribing Information
| Document Title | Year | Source | 
|---|---|---|
| Fabrazyme (agalsidase beta) Prescribing Information. | 2024 | Sanofi-Aventis U.S. LLC., Bridgewater, NJ | 
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source | 
|---|---|---|
| A systematic literature review on the health-related quality of life and economic burden of Fabry disease | 2024 | Orphanet Journal of Rare Diseases | 
| Mechanisms of Neutralizing Anti-drug Antibody Formation and Clinical Relevance on Therapeutic Efficacy of Enzyme Replacement Therapies in Fabry Disease | 2021 | Drugs | 
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 | 

