Formulary
Miglustat: Indications, Dosing, Side Effects and Interactions
When used for Type 1 Gaucher's disease or Niemann-Pick type C disease: Miglustat is an inhibitor of glucosylceramide synthase.
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Miglustat: Indications, Dosing, Side Effects and Interactions
When used for Type 1 Gaucher's disease or Niemann-Pick type C disease: Miglustat is an inhibitor of glucosylceramide synthase.
Drug action
When used for Type 1 Gaucher's disease or Niemann-Pick type C disease: Miglustat is an inhibitor of glucosylceramide synthase. When used for Pompe disease: Miglustat is an enzyme stabiliser of cipaglucosidase alfa.
Indications and dose
**Mild to moderate type I Gaucher's disease**
- **Adult** (By Mouth): for whom enzyme replacement therapy is unsuitable (under expert supervision) 100 mg 3 times a day, reduced if not tolerated to 100 mg 1-2 times a day.
**Treatment of progressive neurological manifestations of Niemann-Pick type C disease (under expert supervision)**
- **Adult** (By Mouth): 200 mg 3 times a day.
**Pompe disease [in combination with cipaglucosidase alfa] (under expert supervision)**
- **Adult** (By Mouth): (body-weight 50 kg and above) 260 mg every 2 weeks, dose to be taken 1 hour (but no more than 3 hours) before the start of cipaglucosidase alfa infusion.
**Treatment of progressive neurological manifestations of Niemann-Pick type C disease (under expert supervision) for miglustat**
- **Child 4-11 years (body surface area up to 0.48 m2)** (By mouth): 100 mg once daily.
- **Child 4-11 years (body surface area 0.48-0.73 m2)** (By mouth): 100 mg twice daily.
- **Child 4-11 years (body surface area 0.74-0.88 m2)** (By mouth): 100 mg 3 times a day.
- **Child 4-11 years (body surface area 0.89-1.25 m2)** (By mouth): 200 mg twice daily.
- **Child 4-11 years (body surface area 1.26 m2 and above)** (By mouth): 200 mg 3 times a day.
- **Child 12-17 years** (By mouth): 200 mg 3 times a day.
Side effects
Common or very common Appetite decreased; asthenia; chills; constipation; depression; diarrhoea; dizziness; flatulence; gastrointestinal discomfort; headache; insomnia; libido decreased; malaise; muscle spasms; muscle weakness; nausea; peripheral neuropathy; sensation abnormal; thrombocytopenia; tremor; vomiting; weight decreased Uncommon Feeling jittery
Interactions
**Other interactions (2):**
- Limited data suggest that co-administration of miglustat and enzyme replacement with imiglucerase in patients with type 1 Gaucher disease may result in decreased exposure to miglustat (approximate...
- This study also indicated that miglustat has no or limited effect on the pharmacokinetics of imiglucerase.
Pregnancy
Avoid (toxicity in animal studies). M
Breast feeding
Avoid (no information available). M
Hepatic impairment
When used for Type 1 Gaucher's disease or Niemann-Pick type C disease: Manufacturer advises caution (no information available).
Renal impairment
When used for Type 1 Gaucher's disease or Niemann-Pick type C disease: Avoid if eGFR less than 30 mL/minute/1.73 m 2 . Dose adjustments For Gaucher's disease initially 100 mg twice daily if eGFR 50-70 mL/minute/1.73 m 2 . Initially 100 mg once daily if eGFR 30-50 mL/minute/1.73 m 2 . For Niemann-Pick type C disease, initially 200 mg twice daily if eGFR 50-70 mL/minute/1.73 m 2 . Initially 100 mg twice daily if eGFR 30-50 mL/minute/1.73 m 2 .
Medicinal forms
Clinical governance
**Author:** MedNext Clinical Team. **Clinical reviewer:** Dr Shameer Deen, MBBS, MS, MRCS. **Sources:** BNF, Indian Pharmacopoeia, CIMS India. **Correction:** Report errors at support@mednext.academy.
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