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Miglustat (Zavesca)
Miglustat inhibits glucosylceramide synthase, reducing the accumulation of glucosylceramide and related glycosphingolipids in cells.
Miglustat inhibits glucosylceramide synthase, reducing the accumulation of glucosylceramide and related glycosphingolipids in cells. Used for Gaucher disease type 1 (substrate reduction therapy), Niemann-Pick type C disease, Progressive myoclonic epilepsy type 2 (Lafora disease) — investigational.
At a glance
| Generic name | Miglustat (Zavesca) |
|---|---|
| Also known as | Zavseca |
| Sponsor | University of Washington |
| Drug class | Glucosylceramide synthase inhibitor |
| Target | Glucosylceramide synthase |
| Modality | Small molecule |
| Therapeutic area | Rare genetic/lysosomal storage diseases |
| Phase | FDA-approved |
Mechanism of action
Miglustat is a glucosylceramide synthase inhibitor that blocks the first committed step in glycosphingolipid synthesis. By reducing glucosylceramide accumulation, it slows the progression of lysosomal storage diseases caused by deficient glucocerebrosidase or galactocerebrosidase activity. This substrate reduction therapy approach helps restore cellular function in affected tissues.
Approved indications
- Gaucher disease type 1 (non-neuronopathic)
- Niemann-Pick disease type C
- Progressive myoclonic epilepsy type 2 (Lafora disease)
Common side effects
- Diarrhea
- Weight loss
- Tremor
- Peripheral neuropathy
- Abdominal pain
- Headache
Key clinical trials
- A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease (PHASE2)
- A Global Prospective Observational Registry of Patients With Pompe Disease
- A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) (PHASE3)
- A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (PHASE3)
- Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation (PHASE2, PHASE3)
- ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD (PHASE3)
- First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (PHASE1, PHASE2)
- Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Miglustat (Zavesca) CI brief — competitive landscape report
- Miglustat (Zavesca) updates RSS · CI watch RSS
- University of Washington portfolio CI