Miglustat Chemical formula
Synonyms: Butyldeoxynojirimycin; n-Butyl-deoxynojirimycin; Miglustaatti; Miglustatum; OGT-918; OXAIDS; SC-48334. 1,5-(Butylimino)1,5-dideoxythyl)piperidine-3,4,5-triol.
Cyrillic synonym: Миглустат.

💊 Chemical information

Chemical formula: C10H21NO4 = 219.3.
CAS — 72599-27-0.
ATC — A16AX06.
ATC Vet — QA16AX06.

💊 Adverse Effects and Precautions

Diarrhoea and other gastrointestinal disturbances, weight loss, tremor, dizziness, headache, cramps, and visual disturbances are frequent in patients receiving miglustat, and some patients may experience paraesthesias, peripheral neuropathy, or thrombocytopenia. Studies in animals have indicated an effect on spermatogenesis; male patients should not attempt conception during, or for 3 months after stopping, treatment. Care is required in renal impairment.

💊 Pharmacokinetics

Miglustat is rapidly absorbed after oral doses with maximum plasma concentrations reached in about 2 hours. It is mainly excreted in the urine with some also excreted in the faeces; the average elimination half-life is 6 to 7 hours.


The average peak plasma concentration was reduced by 37% when miglustat was taken with food by 24 healthy subjects. However, there was no clinically significant effect on the extent of absorption (area under the curve was decreased by 14%).1 Licensed product information states that miglustat may be taken with or without food.
1. van Giersbergen PLM, Dingemanse J. Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase. J Clin Pharmacol 2007; 47: 1277–82.

💊 Uses and Administration

Miglustat is an inhibitor of the enzyme glucosylceramide synthase, responsible for the first step in the synthesis of glucosylceramide and most other glycolipids. It is used to help prevent the accumulation of glucosylceramide in patients with mild to moderate type 1 Gaucher disease who cannot be treated with enzyme replacement therapy. The initial dose is 100 mg orally 3 times daily; reduction to 100 mg once or twice daily may be necessary in some patients because of diarrhoea. For details of reduced doses in patients with renal impairment, see below. Miglustat has also been used for the treatment of Niemann-Pick disease, type C.
1. McCormack PL, Goa KL. Miglustat. Drugs 2003; 63: 2427–34
2. Weinreb NJ, et al. Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Am J Hematol 2005; 80: 223–9
3. Giraldo P, et al. Short-term effect of miglustat in every day clinical use in treatment-naïve or previously treated patients with type 1 Gaucher’s disease. Haematologica 2006; 91: 703–6
4. Elstein D, et al. Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement. Blood 2007; 110: 2296–2301
5. Patterson MC, et al. Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet Neurol 2007; 6: 765–72.

Administration in renal impairment.

The initial dose of miglustat should be reduced in renal impairment according to the patient’s creatinine clearance (CC):
CC 50 to 70 mL/minute per 1.73 m2: 100 mg twice daily
CC 30 to 50 mL/minute per 1.73 m2: 100 mg daily
CC less than 30 mL/minute per 1.73 m2: not recommended

💊 Preparations

Proprietary Preparations

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Published May 08, 2019.