Drug updated on 11/4/2024
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 |