miglustat
Orphan DrugFDA Approved, EMA Approved
Description
Miglustat is a small molecule iminosugar that acts as a glucosylceramide synthase inhibitor, reducing the synthesis of glycosphingolipids. It is used as substrate reduction therapy for lysosomal storage disorders affecting sphingolipid metabolism. The drug provides an alternative treatment approach when enzyme replacement therapy is not suitable or available.
Indications & Therapeutic Use
Gaucher disease type 1, Niemann-Pick disease type C, disorders of sphingolipid biosynthesis
Global Availability (9 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
miglustat
| Generic Name | miglustat |
| Brands | 1 brand available |
| Active Ingredient | miglustat |
| Drug Class | Gaucher disease type 1 |
| Manufacturer | Actelion Pharmaceuticals |
| Dosage Forms | Oral capsule, 100mg |
| Medical Code | A16AX06 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 14 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT00358150 |
| Countries | 9 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations10 Validated Nodes