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Class: Other Miscellaneous Therapeutic Agents
VA Class: HS900
Chemical Name: 1-Butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
Molecular Formula: C10H21NO4
CAS Number: 72599-27-0
Brands: Zavesca


Glucosylceramide synthase (ceramide glucosyltransferase) inhibitor; decreases glucocerebroside accumulation in macrophages and some manifestations (hepatomegaly, splenomegaly) of type 1 Gaucher’s disease.1 15 19 20 21

Uses for Miglustat

Gaucher’s Disease

Management of mild to moderate nonneuronopathic (type 1) Gaucher’s disease in adults for whom enzyme replacement therapy is unsuitable (e.g., because of allergy, hypersensitivity, poor venous access).1

Safety and efficacy not established in patients with severe type 1 Gaucher’s disease (i.e., hemoglobin <9 g/dL, platelet count <50,000/mm3, or active bone disease).1

Designated an orphan drug by the FDA for the management of Gaucher’s disease.18

Enzyme replacement therapy with alglucerase or imiglucerase is current treatment of choice for type 1 Gaucher’s disease.1 2 3 4 6 7 11 12 13 14 16

Miglustat Dosage and Administration


  • If a dose is missed, that dose should be skipped and the next dose taken at the usual time.1

  • Reduced dosage may be necessary in some patients because of adverse effects (e.g., diarrhea, tremor).1 16


Oral Administration

Administer 3 times daily at regular intervals, without regard to meals.1



Gaucher’s Disease

100 mg 3 times daily.1 16

Reduce dosage to 100 mg once or twice daily if necessary because of adverse effects.1 16

Special Populations

Renal Impairment

Dosage in Renal Impairment

Clcr (mL/min)

Initial dosage


100 mg twice daily1


100 mg once daily1


Use not recommended1

Geriatric Patients

Select dosage with caution because of possible age-related decreases in renal, hepatic, and/or cardiac function and concomitant disease and drug therapy.1

Cautions for Miglustat


  • Known hypersensitivity to miglustat or any ingredient in the formulation.1

  • Women who are or may become pregnant.1



Peripheral Neuropathy

Peripheral neuropathy reported; monitor all patients with neurologic evaluation at baseline and every 6 months while on therapy.1

If symptoms (e.g., numbness, tingling) occur, reassess risk versus benefit of therapy; consider discontinuance.1

General Precautions

Therapy should be directed by clinicians knowledgeable in the management of Gaucher’s disease.1


Tremor or exaggerated physiologic hand tremor reported in about 30% of patients.1 24

Usually began within the first month, often resolved between month 1 and 3 of treatment.1

Dosage reduction may ameliorate tremor, usually within days; drug discontinuance occasionally necessary.1

Diarrhea and Weight Loss

Diarrhea in approximately 85% of patients studied; associated with increased use of antidiarrheal drugs, usually loperamide.1 24

Apparently osmotic in nature, resulting from inhibition of disaccharidase; incidence decreases over time with continued use.1

Patients should avoid foods with high carbohydrate content.1

Weight loss in up to 65% of patients studied, generally during first 12 months of therapy.1

Weight loss may result from diarrhea and associated GI complaints, decreased food intake, or a combination of these and other factors.1

Effect on Fertility in Males

Adversely affects spermatogenesis and sperm parameters; decreases fertility (rats).1

All males should use effective birth control while on therapy.1

Before attempting conception, discontinue therapy and maintain effective contraceptive methods for 3 months.1

Specific Populations


Category X.1 (See Contraindications under Cautions.)


Not known whether miglustat is distributed into milk.1 Discontinue nursing or the drug.1

Pediatric Use

Safety and efficacy not established in children <18 years of age.1

Geriatric Use

Insufficient experience in patients ≥65 years of age to determine whether geriatric patients respond differently from younger adults; select dosage with caution.1

Renal Impairment

Dosage adjustments necessary based on degree of renal impairment; do not use in severe renal impairment. (See Renal Impairment under Dosage and Administration.) 1

Common Adverse Effects

Diarrhea, flatulence, abdominal pain, abdominal distension with or without gas, nausea, vomiting, bloating, anorexia, dyspepsia, epigastric pain (not related to food), constipation, dry mouth, weight loss, headache, tremor, dizziness, unsteady gait, leg cramps, cramps, back pain, paresthesia, heaviness in limbs, generalized weakness, migraine, visual disturbance, memory loss, thrombocytopenia, menstrual disorder.1 24

Interactions for Miglustat

Drugs Metabolized by Hepatic Microsomal Enzymes

No evidence of metabolism in humans; pharmacokinetic interactions unlikely.1

Specific Drugs





Possible decreased plasma imiglucerase concentrations 1

Possible decreased peak plasma miglustat concentrations (22%) and AUC (14%) 1

Concomitant use increased imiglucerase clearance by 70%; results inconclusive (small number of patients studied, variable imiglucerase dosages)1

Effect on miglustat pharmacokinetics not considered clinically important1

Combination use not indicated1


Pharmacokinetic interaction unlikely1

Concomitant administration apparently did not substantially alter pharmacokinetics of miglustat1

Miglustat Pharmacokinetics



Food decreases the rate but not the extent of absorption.1



Distributed into extravascular tissues.1

Not known whether miglustat crosses the placenta or is distributed into milk.1

Plasma Protein Binding

Does not bind to plasma proteins.1



No evidence of metabolism in humans.1

Elimination Route

Excreted unchanged in urine.1 15


6–7 hours.1 15

Special Populations

In patients with mild, moderate, or severe renal impairment, miglustat clearance is reduced by approximately 40, 60, or ≥70%, respectively.1 15





20–25°C (may be exposed to 15–30°C).1


  • N-alkylated imino sugar; competitive and reversible inhibitor of ceramide glucosyltransferase (glucosylceramide synthase), which catalyzes the formation of glucosylceramide (glucocerebroside).1 15

  • Inhibits the formation of glucocerebroside, substrate for glucocerebrosidase enzyme deficient in type 1 Gaucher’s disease.1 15

  • Substrate reduction therapy with miglustat allows residual activity of deficient endogenous glucocerebrosidase to be more effective, decreasing glucocerebroside accumulation in macrophages;1 15 19 20 decreases hepatomegaly and splenomegaly but has limited effect on increasing hemoglobin and platelet counts in patients with type 1 Gaucher’s disease.1 15 21

  • Actions are unlike those of enzyme replacement therapy, which increases glucocerebroside degradation in macrophages to reduce manifestations of Gaucher’s disease.1 2 3 4 6 7 11 12 13 14 15 16

  • In type 1 Gaucher’s disease, lipid-engorged macrophages infiltrate the viscera (especially liver and spleen), lymph nodes, and bone marrow, resulting in lipidosis and clinical manifestations of hepatosplenomegaly, anemia, thrombocytopenia, and bone lesions in severe disease.2 3 6 7 8 9 10 13 15

Advice to Patients

  • Importance of informing patients of risks and benefits of miglustat therapy, and about alternative therapy (e.g., enzyme replacement therapy).1

  • Importance of advising patients that diarrhea and other adverse GI effects and weight loss are common, and to adhere to dietary instructions.1

  • Importance of informing clinician of any numbness, pain, or burning in the hands or feet and of the occurrence or worsening of tremor.1

  • Importance of women informing their clinician if they are or plan to become pregnant or plan to breast-feed; necessity for clinicians to advise women to avoid pregnancy during therapy and, if pregnancy occurs, to advise woman of risk to the fetus.1

  • Importance of men using effective birth control methods during miglustat use and for 3 months following discontinuance of the drug.1

  • Importance of informing clinicians of existing or contemplated concomitant therapy, including prescription and OTC drugs and dietary and herbal supplements, as well as any concomitant illnesses.1

  • Importance of informing patients of other important precautionary information. (See Cautions.)


Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.



Dosage Forms


Brand Names




100 mg



AHFS DI Essentials. © Copyright 2017, Selected Revisions August 1, 2005. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.


1. Actelion Pharmaceuticals. Zavesca (miglustat) capsules prescribing information. South San Francisco, CA; 2003 Jul 31.

2. Beutler E. Gaucher’s disease. N Engl J Med. 1991; 325:1354-60. [PubMed 1922238]

3. Anon. Alglucerase for Gaucher’s disease. Med Lett Drugs Ther. 1991; 33:82. [PubMed 1865852]

4. Elstein D, Abrahamov A, Hadas-Halpern I et al. Low-dose low-frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type I Gaucher disease. QJM. 1998; 91:483-8. [PubMed 9797931]

5. Elstein D, Abrahamov A, Hadas-Halpern I et al. Gaucher’s Disease. Lancet. 2001; 358:324-7. [PubMed 11498237]

6. Genzyme Corporation. Cerezyme (imiglucerase) prescribing information. Cambridge, MA; 2003 Mar.

7. Barton NW, Furbish FS, Murray GJ et al. Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease. Proc Natl Acad Sci USA. 1990; 87:1913-6. [PubMed 2308952]

8. Brady RO, Kanfer JN, Shapiro D. Metabolism of glucocerebrosides: II. Evidence of an enzymatic deficiency in Gaucher’s disease. Biochem Biophys Res Commun. 1965; 18:221-5. [PubMed 14282020]

9. Britton DE, Leinikki PO, Barranger JA et al. Gaucher’s disease: lack of antibody response to intravenous glucocerebrosidase. Life Sci. 1978; 23:2517-20. [PubMed 732537]

10. Basu A, Prence E, Garrett K et al. Comparison of N-acyl phosphatidylethanolamines with different N-acyl groups as activators of glucocerebrosidase in various forms of Gaucher’s disease. Arch Biochem Biophys. 1985; 243:28-34. [PubMed 3933429]

11. Genzyme Corporation. Ceredase (alglucerase) prescribing information. Cambridge, MA; 1992 Feb.

12. Reviewers’ comments (personal observations) on alglucerase.

13. Weinreb NJ, Charrow J, Andersson HC et al. Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Register. Am J Med. 2002; 113:112-9. [PubMed 12133749]

14. Niederau C, Haussinger D. Gaucher’s disease: a review for the internist and hepatologist. Hepatogastroenterology. 2000; 47:984-7. [PubMed 11020862]

15. McCormack PL, Goa KL. Miglustat. Drugs. 2003;63:2427-34.

16. Grabowski GA, Barton NW, Pastores G et al. Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase form natural and recombinant sources. Ann Intern Med. 1995; 122:33-9. [PubMed 7985893]

17. Genzyme Corporation, Cambridge, MA. Personal observation on alglucerase.

18. Food and Drug Administration. Cumulative list of orphan drugs designated and/or approved. Rockville, MD; 2004, Aug 31. From FDA web site (

19. Lachmann RH, Platt FM. Substrate reduction therapy for glycosphingolipid storage disorders. Expert Opin Invest Drugs. 2001; 10:455-66.

20. Pastores GM, Barnett NL. Substrate reduction therapy: miglustat as a remedy for symptomatic patients with Gaucher disease type 1. Expert Opin Invest Drugs. 2003; 23:273-81.

21. Cox T, Lachmann R, Hollak C et al. Novel oral treatment of Gaucher’s disease with N-butyldeoxynojirimycin. (OGT 918) to decrease substrate biosynthesis. Lancet. 2000; 355:1481-5. [PubMed 10801168]

22. Heitner R, Elstein D, Aerts J et al. Low-dose N-butyldeoxynojirimycin. (OGT 918) for type 1 Gaucher disease. Blood Cells Mol Dis. 2002; 28:127-33. [PubMed 12064906]

23. Oxford GlycoSciences Plc. Oxford GlycoSciences Plc swith/combination study 004 results on Vevesca (OGT 918) presented at EWGGD [media release]. 2002 May 2.

24. Actelion. South San Francisco, CA: Personal communication.