Glucosylceramide synthase inhibitor
Miglustat
Brand names: Zavesca
Adult dose
Dose: 100mg PO TDS for Gaucher type 1; 200mg PO TDS for Niemann-Pick C
Route: PO
Frequency: TDS
Clinical pearls
- Type 1 Gaucher disease (mild-moderate, where ERT unsuitable); Niemann-Pick disease type C
- Specialist metabolic
Contraindications
- Severe renal impairment
- Pregnancy/breastfeeding
- Hypersensitivity
Side effects
- Diarrhoea (common — adjust diet)
- Tremor
- Weight loss
- Peripheral neuropathy
- Thrombocytopenia
Monitoring
- Neurological exam
- FBC
- Weight
Reference: BNF; NICE; SmPC; https://bnf.nice.org.uk/drugs/miglustat/. Verify against your local formulary and the latest BNF before prescribing.
Related
Curated clinical cross-links plus same-class fallbacks.
Calculators
- SMART Risk Score for Recurrent CVD · Cardiovascular Risk
- PCSK9 Inhibitor Eligibility Assessment · Lipid Management
- Immune-Related Adverse Events (irAE) -- GI Toxicity Colitis Grading · Oncology-Related GI
- irAE Hepatitis Grading (CTCAE) · Immunotherapy
- DIPSS — Dynamic International Prognostic Scoring System for Myelofibrosis · Cancer Prognosis
- BALL Score for Relapsed/Refractory CLL · Leukaemia
Pathways
- Diabetic Ketoacidosis (DKA) · JBDS 2013 / Joint British Diabetes Societies; NICE NG17
- Adult Hypoglycaemia (Treated Diabetes) · JBDS-IP (2023): Hospital Management of Hypoglycaemia
- Adrenal Crisis · Society for Endocrinology Emergency Guidance (2024)
- Type 2 Diabetes Management · NICE NG28 2022
- Hyperthyroidism Management · BTA / ETA 2018
- Adrenal Insufficiency · Society of Endocrinology / ESE 2016