Drug updated on 4/16/2024
| 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.
Summary
- Eliglustat (Cerdelga) is indicated for the long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers as detected by an FDA-cleared test.
- The information was derived from a systematic review/meta-analysis on the treatment of Gaucher disease Type 1.
- Eliglustat's effectiveness and safety profile might be influenced by the patient's CYP2D6 metabolizer status, which dictates how well the drug is processed in their body.
- The study suggests that eliglustat can maintain or potentially improve clinical parameters in patients previously treated with other therapies for Gaucher disease, making it an effective option for ongoing management of Gaucher disease type 1.
- Compared to Enzyme Replacement Therapies (ERTs), it was noted that another therapy did not show significant improvement in blood outcomes for naïve patients and was associated with a decrease in platelet levels, suggesting limitations in its effectiveness and potential safety concerns compared to eliglustat.
- It is important to match the choice of therapy such as eliglustat based on the specific clinical profile of each patient, including their CYP2D6 metabolizer status, to maximize therapeutic benefits while minimizing potential risks.
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. | 2022 | 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 |