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Miglustat (Gaucher Disease) (Monograph)

Brand name: Zavesca
Drug class:

Medically reviewed by on Apr 10, 2024. Written by ASHP.


Glucosylceramide synthase (ceramide glucosyltransferase) inhibitor.

Uses for Miglustat (Gaucher Disease)

Gaucher Disease

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

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

Miglustat (Gaucher Disease) Dosage and Administration



Oral Administration

Administer orally 3 times daily at regular intervals.

If a dose is missed, skip dose and take next dose at usual time.



Gaucher Disease

100 mg 3 times daily.

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

Special Populations

Renal Impairment

Dosage in Renal Impairment

Clcr (mL/min)

Initial dosage


100 mg twice daily


100 mg once daily


Use not recommended

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.

Cautions for Miglustat (Gaucher Disease)




Peripheral Neuropathy

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

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


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

Usually develops within the first month, often resolving between 1–3 months of treatment.

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

Diarrhea and Weight Loss

Diarrhea reported in approximately 85% of patients. Apparently osmotic in nature, resulting from inhibition of disaccharidase; incidence decreases over time with continued use.

Patients should avoid foods with high carbohydrate content.

Weight loss occurred in up to 65% of patients. May result from diarrhea and associated GI complaints, decreased food intake, or a combination of these and other factors.

If persistent GI symptoms occur that do not respond to usual interventions, evaluate for presence of underlying GI disease. Weigh risks versus benefits of continued treatment in patients with substantial GI disease (e.g., inflammatory bowel disease).

Reduction in Platelet Counts

Mild reductions in platelet counts reported; not associated with bleeding.

Monitor platelet counts.

Specific Populations


Category C.

No adequate and well-controlled studies in pregnant women. Animal studies suggest that the drug may cause fetal harm. Use during pregnancy only if potential benefit justifies potential risk to fetus.


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

Pediatric Use

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

Geriatric Use

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

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.)

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.

Drug Interactions

Drugs Metabolized by Hepatic Microsomal Enzymes

Does not inhibit CYP1A2, 2A6, 2C9, 2C19, 2D6, 2E1, 3A4, and 4A11 isoenzymes in vitro; clinically important drug interactions unlikely with drugs metabolized by these isoenzymes.

Specific Drugs





Increased imiglucerase clearance by 70%; however, results inconclusive (small number of patients studied, variable imiglucerase dosages)

Pharmacokinetics of miglustat not substantially altered


Pharmacokinetic interaction unlikely; pharmacokinetics of miglustat not substantially altered

Miglustat (Gaucher Disease) Pharmacokinetics



Food decreases the rate but not the extent of absorption.



Distributed into extravascular tissues.

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

Plasma Protein Binding

Does not bind to plasma proteins.


Elimination Route

Excreted unchanged in urine.


6–7 hours.

Special Populations

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





20–25°C (may be exposed to 15–30°C).


Advice to Patients

Additional Information

The American Society of Health-System Pharmacists, Inc. represents that the information provided in the accompanying monograph was formulated with a reasonable standard of care, and in conformity with professional standards in the field. Readers are advised that decisions regarding use of drugs are complex medical decisions requiring the independent, informed decision of an appropriate health care professional, and that the information contained in the monograph is provided for informational purposes only. The manufacturer’s labeling should be consulted for more detailed information. The American Society of Health-System Pharmacists, Inc. does not endorse or recommend the use of any drug. The information contained in the monograph is not a substitute for medical care.


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 2024, Selected Revisions April 10, 2024. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.

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