Overview
Eliglustat is a glucosylceramide synthase inhibitor used for the long-term treatment of type 1 Gaucher disease. Gaucher disease is a rare genetic disorder characterized by the deficiency of acid β-glucosidase, an enzyme that converts glucosylceramide into glucose and ceramide. In patients with Gaucher disease, the accumulation of glucosylceramide leads to the formation of Gaucher cells that infiltrate the liver, spleen, bone marrow and other organs. This leads to complications such as anemia and thrombocytopenia. By inhibiting glucosylceramide synthase, eliglustat reduces the accumulation of glucosylceramide. Eliglustat is mainly metabolized by CYP2D6. Patients selected for eliglustat treatment undergo an FDA-cleared genotyping test to establish if they are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs). The results of this test dictate eliglustat dosing recommendations for each type of patient. There are no dosing recommendations for CYP2D6 ultra-rapid or indeterminate metabolizers. Eliglustat was approved by the FDA in August 2014 as an oral substrate reduction therapy for the first-line treatment of type 1 Gaucher disease. Enzyme replacement continues to be the standard of care for the treatment of type 1 Gaucher disease (imiglucerase, velaglucerase alfa, taliglucerase alfa); however, oral substrate reduction therapies with favourable safety profiles, such as eliglustat, represent a treatment alternative.
Indication
Eliglustat is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease in adult patients who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. CYP2D6 ultra-rapid metabolizers may not achieve adequate eliglustat concentrations to achieve a therapeutic effect. A specific dosage cannot be recommended for CYP2D6 indeterminate metabolizers.
Associated Conditions
- Gaucher Disease, Type 1
Research Report
Eliglustat (DB09039): A Comprehensive Pharmacological and Clinical Monograph
Section 1: Drug Profile and Chemical Characteristics
Eliglustat is a small molecule therapeutic agent developed as a substrate reduction therapy for a rare genetic disorder. Its pharmacological activity, clinical utility, and safety profile are intrinsically linked to its specific chemical structure and physicochemical properties. This section provides a foundational overview of its nomenclature, chemical identity, and structural features that define its function as a targeted enzyme inhibitor.
1.1 Nomenclature and Key Identifiers
For unambiguous identification across scientific, clinical, and regulatory domains, Eliglustat is cataloged under a variety of names and unique identifiers.
- Generic Name: The officially recognized non-proprietary name for the active substance is Eliglustat.[1]
- Brand Name: It is marketed globally under the trade name Cerdelga.[1]
- Drug Type: Eliglustat is classified as a Small Molecule drug [User Query].
- Developmental and Research Synonyms: During its development by Genzyme, it was referred to by codes such as GENZ-112638 and Genz-99067.[7]
- Systematic (IUPAC) Name: The precise chemical structure is defined by its IUPAC name: N-octanamide.[1]
- Database and Registry Identifiers: To ensure global standardization, Eliglustat is assigned unique codes in major chemical and pharmacological databases. These are essential for literature searches, regulatory tracking, and bioinformatics. A summary of these identifiers is provided in Table 1.
1.2 Physicochemical Properties and Formulation
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/07/29 | Not Applicable | Not yet recruiting | Chinese PLA General Hospital | ||
2024/07/26 | Phase 2 | Not yet recruiting | |||
2024/01/05 | Phase 1 | Completed | |||
2024/01/03 | Phase 1 | Completed | |||
2023/11/22 | Phase 1 | Completed | |||
2021/06/30 | Phase 1 | Completed | Chinese PLA General Hospital | ||
2018/05/09 | Not Applicable | Completed | |||
2018/04/02 | Phase 3 | Active, not recruiting | |||
2015/09/01 | Phase 1 | Completed | |||
2015/09/01 | Phase 1 | Completed | Genzyme, a Sanofi Company |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Genzyme Corporation | 58468-0220 | ORAL | 84 mg in 1 1 | 3/3/2024 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
| No EMA approvals found for this drug. | |||
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No HSA approvals found for this drug. | |||||
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| CERDELGA eliglustat (as tartrate) 84 mg capsule blister pack | 218172 | Medicine | A | 2/17/2015 |
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
|---|---|---|---|---|---|
| No Health Canada approvals found for this drug. | |||||
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
|---|---|---|---|---|---|
| CERDELGA 84 MG CAPSULAS DURAS | 114974001 | CÁPSULA DURA | Uso Hospitalario | Commercialized |
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Philippines FDA approvals found for this drug. | |||||
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Saudi SFDA approvals found for this drug. | |||||
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Malaysia NPRA approvals found for this drug. | |||||
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
|---|---|---|---|---|---|
| No UK EMC drug information found for this drug. | |||||
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