Drug updated on 4/17/2024
| 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.
Summary
- Agalsidase beta (Fabrazyme) is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease, an X-linked inheritable disorder characterized by deficient activity of α-galactosidase leading to progressive lipid metabolism disorders.
- Two studies were reviewed which provided a detailed summary and comparison of the effects and safety of agalsidase beta in Enzyme Replacement Therapy (ERT) for Fabry disease compared to other treatments such as agalsidase alfa.
- The first study highlights challenges in evaluating ERT outcomes due to a scarcity of randomized, placebo-controlled clinical trials. It emphasizes heterogeneity in patient populations, outcomes, and ERT regimens across studies, necessitating unsystematic literature reviews.
- The second study focuses on effectiveness specifically among adult male patients, revealing potential benefits including a significant decrease in globotriaosylceramide levels across various biomarkers and organ systems; slowing the decline in renal function; reducing or stabilizing cardiac abnormalities; and improving neurological and gastrointestinal symptoms along with quality-of-life outcomes.
- While both agalsidase beta (Fabrazyme) and alfa are used for treating Fabry disease, the studies primarily focus on agalsidase beta without explicitly stating one as superior to another but hinting at potential differences between the two regarding their effectiveness and dosage requirements.
- Although safety isn't explicitly detailed, it's underscored that early initiation before organ damage occurs can be beneficial. Patient-specific factors such as age, genotype, disease severity, and level of organ involvement play a crucial role in determining treatment outcome, suggesting the need for a tailored therapeutic approach based on individual characteristics.
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 |