Drug updated on 12/11/2024
| Dosage Form | Capsule (oral: 84 mg) |
| Drug Class | Glucosylceramide synthase inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.
Latest News
Summary
- This summary is based on the review of one systematic review/meta-analysis. [1]
- Eliglustat for Gaucher Disease: Eliglustat demonstrated stable outcomes in experienced Gaucher disease (GD) patients, maintaining health status in those previously treated with other therapies.
- Effectiveness of Enzyme Replacement Therapies (ERTs): ERTs, including imiglucerase, velaglucerase alfa, and taliglucerase alfa, were effective in both naive (untreated) and experienced GD patients, supporting their broad applicability across treatment backgrounds.
- Miglustat Outcomes: Miglustat showed limited effectiveness, with no significant improvements in blood outcomes in naive patients and a reduction in platelet levels in experienced patients.
- 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 |
|---|---|---|
| Cerdelga (eliglustat) Prescribing Information. | 2024 | Sanofi |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| A Systematic Review and Meta-analyses of Longitudinal Studies on Drug Treatments for Gaucher Disease | 2023 | Annals of Pharmacotherapy |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Recommendations for oral treatment for adult patients with type 1 Gaucher disease. | 2022 | Revista Clínica Española |