Eliglustat
Orphan DrugFDA Approved, EMA Approved
Contents
Description
Eliglustat is a selective glucosylceramide synthase inhibitor that reduces the accumulation of glucosylceramide in cells. It represents a substrate reduction therapy for specific lysosomal storage disorders affecting lipid metabolism. The drug is administered orally and requires CYP2D6 genotyping prior to initiation.
Indications & Therapeutic Use
Gaucher disease type 1, disorders of sphingolipid metabolism
Linked Diseases:
Global Availability (7 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Eliglustat
| Generic Name | eliglustat |
| Brands | 1 brand available |
| Active Ingredient | eliglustat tartrate |
| Drug Class | Gaucher disease type 1 |
| Manufacturer | Sanofi Genzyme |
| Dosage Forms | Oral capsule, 84mg |
| Medical Code | A16AX09 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 10 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT00358150 |
| Countries | 7 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations8 Validated Nodes