Drug updated on 12/11/2024
| Dosage Form | Injection (intravenous; 200 units/vial) |
| Drug Class | Hydrolytic lysozomal glucocerebroside-specific enzyme |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease.
Latest News
Summary
- This summary is based on the review of one systematic review/meta-analysis. [1]
- Taliglucerase alfa demonstrated effectiveness as an enzyme replacement therapy for Gaucher disease in both naive and experienced patients, with outcomes comparable to imiglucerase and velaglucerase alfa.
- Miglustat showed limited efficacy, with no significant blood outcome improvements in naive patients and a reduction in platelet levels in experienced patients, making it less favorable compared to taliglucerase alfa and other enzyme replacement therapies.
- Eliglustat maintained stable outcomes in experienced patients, indicating effectiveness, though its impact was primarily assessed in this group.
- There is no safety information available in the reviewed studies.
- There is no population types or subgroups information available in the reviewed studies.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Elelyso (taliglucerase alfa) Prescribing Information. | 2024 | Pfizer Inc., New York, NY |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| A Systematic Review and Meta-analyses of Longitudinal Studies on Drug Treatments for Gaucher Disease | 2023 | The Annals of Pharmacotherapy |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Management goals of type 1 Gaucher disease in South Africa: an expert Delphi consensus document on good clinical practice. | 2023 | PLoS One |