Section 12 · Monograph
Miglustat
Reviewed Nov 2026FDA label
Zavesca / YargesaGlucosylceramide synthase inhibitor
- Forms
- Cap: 100mg
- Dose
- Niemann-Pick type C (off-label in US): adults 200mg three times daily; pediatric dosing by body surface area | Gaucher type 1: 100mg three times daily
- MOA
- Substrate reduction of glycosphingolipid synthesis
Indications
- Mild to moderate type 1 Gaucher disease in adults for whom enzyme replacement therapy is not an option (US labeling)FDA-labeled
Off-Label Uses
- Niemann-Pick disease type C (approved in some regions; used in combination with arimoclomol in the US)Off-label
Mechanism of Action
- Reversible inhibitor of glucosylceramide synthase, reducing synthesis of glycosphingolipids (substrate reduction therapy)
Full Dosing
Adult / usual
- Gaucher type 1: 100mg orally three times daily
Renal
- Reduce dose in renal impairment (CrCl under 70mL/min)
Pediatric
- NPC (off-label in the US): adults 200mg three times daily; pediatric dosing is by body surface area
Contraindications
- Hypersensitivity to miglustat or its components
Drug Interactions
- Other drugs causing diarrhea or neuropathy
- Do not combine with Opfolda (a different miglustat product) as dosed for Pompe disease
Adverse Effects
- Diarrhea, flatulence, abdominal pain
- Weight loss
- Tremor and peripheral neuropathy
- Thrombocytopenia
Monitoring Parameters
- Neurologic exam including tremor and neuropathy
- Platelet count and vitamin B12
- Weight and gastrointestinal tolerance
Pregnancy & Lactation
- Avoid in pregnancy; effective contraception is advised
- Not recommended during breastfeeding