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Medically reviewed by Last updated on Jul 25, 2020.


(MIG loo stat)

Index Terms

  • OGT-918

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Capsule, Oral:

Zavesca: 100 mg [contains soybean lecithin]

Generic: 100 mg

Brand Names: U.S.

  • Zavesca

Pharmacologic Category

  • Enzyme Inhibitor
  • Glucosylceramide Synthase Inhibitor


Miglustat competitively and reversibly inhibits the enzyme needed to produce glycosphingolipids and decreases the rate of glycosphingolipid glucosylceramide formation. Glucosylceramide accumulates in type 1 Gaucher disease, causing complications specific to this disease.


Vd: 83-105 L


No evidence of metabolism in humans


Urine (as unchanged drug)

Time to Peak

Plasma: 2-2.5 hours

Half-Life Elimination

6-7 hours

Protein Binding

No binding to plasma proteins

Special Populations: Renal Function Impairment

Limited data suggests that the clearance of miglustat decreases 40% and 60% with mild and moderate renal impairment, respectively. A decreased clearance of 70% has been suggested in patients with severe renal impairment.

Use: Labeled Indications

Gaucher disease: Treatment of adult patients with mild-to-moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (eg, due to allergy, hypersensitivity, or poor venous access)

Off Label Uses

Niemann-Pick Type C disease

Data from a randomized, controlled trial supports the use of miglustat in the treatment of Niemann-Pick Type C disease. Additional trials may be necessary to further define the role of miglustat in this condition [Patterson 2007].

Based on the Niemann-Pick Type C Guidelines Working Group, miglustat is an effective and recommended treatment option in the management of existing neurologic manifestations of Niemann-Pick Type C disease [Patterson 2012].


There are no contraindications listed in the manufacturer’s labeling.

Canadian labeling: Hypersensitivity to miglustat or any component of the formulation; women who are or may become pregnant

Dosing: Adult

Type 1 Gaucher disease: Oral: 100 mg 3 times daily; dose may be reduced to 100 mg 1 to 2 times daily in patients with adverse effects (ie, tremor, diarrhea)

Niemann-Pick Type C disease (off-label use) Oral: 200 mg 3 times daily (Patterson 2007; Santos-Lozano 2015)

Dosing: Geriatric

Refer to adult dosing.

Dosing: Pediatric

Niemann-Pick Type C disease: Limited data available (Heron 2012; Patterson 2012): Note: Therapy is recommended for patients with neurological, cognitive, or psychiatric disease manifestations (Patterson 2012). Infant data is very limited; reported infant neurological effects (aside from seizures) have included hypotonia, dysphagia, feeding difficulties (onset: 5 to 12 months of age); earliest reported miglustat therapy is 7 months of age (DiRoccoa 2012; Heron 2012). Doses may be initiated slowly and adjusted for tolerability, particularly GI effects (diarrhea which may require dietary and pharmacologic management (Heron 2012; Patterson 2012). Several months of therapy may be necessary to see clinical benefit (eg, 6 to 12 months) (Patterson 2012).

Infants and Children <12 years: Oral:

BSA ≤0.47 m2: 100 mg once daily

BSA >0.47 to 0.73 m2: 100 mg twice daily

BSA >0.73 to 0.88 m2: 100 mg 3 times daily

BSA >0.88 to 1.25 m2: 200 mg twice daily

BSA >1.25 m2: 200 mg 3 times daily

Children ≥12 years and Adolescents: 200 mg 3 times daily (Zavesca prescribing information [Canada 2012]; Zavesca prescribing information [European Medicines Agency 2012])


May be administered with or without food; administration between meals may decrease the incidence of diarrhea. Capsules should be taken at the same time each day at regular intervals. If patient is unable to tolerate or swallow capsule whole and powder is administered, mix powder into a liquid immediately prior to use (do not store); sweetening agents are not expected to interact (data on file [Actelion Pharmaceuticals Ltd, 2011])

Dietary Considerations

Patients with diarrhea should avoid foods with high carbohydrate content.


Store at 20°C to 25°C (68°F to 77°F). Brief exposure to 15°C to 30°C (59°F to 86°F) permitted. Note: Extended storage information may be available; contact product manufacturer to obtain current recommendations.

Drug Interactions

There are no known significant interactions.

Adverse Reactions

The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.

Percentages reported from open-label, uncontrolled monotherapy trials.


Central nervous system: Headache (21% to 22%), dizziness (≤11%)

Endocrine & metabolic: Weight loss (39% to 67%)

Gastrointestinal: Diarrhea (89% to 100%), abdominal pain (18% to 67%), flatulence (29% to 50%), nausea (8% to 22%), vomiting (4% to 11%)

Neuromuscular & skeletal: Tremor (11% to 30%), weakness (17%), leg cramps (4% to 11%)

Ophthalmic: Visual disturbance (≤17%)

1% to 10%:

Central nervous system: Memory impairment (8%), unsteady gait (8%), paresthesia (≤7%), migraine (≤6%)

Endocrine & metabolic: Menstrual disease (≤6%)

Gastrointestinal: Abdominal distension (8%), constipation (8%), xerostomia (8%), anorexia (≤7%), dyspepsia (≤7%), bloating (≤6%), epigastric pain (≤6%)

Hematologic & oncologic: Thrombocytopenia (6% to 7%)

Neuromuscular & skeletal: Back pain (8%)


Concerns related to adverse effects:

• Diarrhea: Observed in the majority of patients, many also reported weight loss (within first 12 months of treatment). Diarrhea decreased over time with continued treatment, and may respond to diet modification (eg, reduction of sucrose, lactose and other carbohydrate intake), taking miglustat between meals, and/or to anti-diarrheal medications. If diarrhea occurs during treatment, instruct patients to avoid foods with high carbohydrate content. If symptoms persist, evaluate patients for underlying GI disease.

• Peripheral neuropathy: Peripheral neuropathy has been reported; neurologic monitoring is required. Weigh risk versus benefit of therapy if patient develops symptoms (eg, numbness and tingling); treatment discontinuation may be necessary.

• Platelet counts decreased: Mild decrease in platelet counts (without bleeding) has been observed in patients with type 1 Gaucher disease switched from enzyme replacement therapy; monitor platelet counts during therapy.

• Tremor: New-onset or exacerbations of existing tremor may occur. Tremor typically begins within the first month of treatment and may resolve over time (1-3 months) or respond to dosage reduction. Treatment discontinuation may be necessary if tremor does not respond within days of dose reduction.

Disease-related concerns:

• Renal impairment: Use with caution in patients with renal impairment; dosage adjustments recommended. Not recommended in patients with severe impairment.

Other warnings/precautions:

• Experienced physician: Should be administered under the supervision of a physician experienced in treatment of Gaucher disease.

• Registry: A registry has been established and all patients with Gaucher disease, and physicians who treat Gaucher disease are encouraged to participate. Information on the International Collaborative Gaucher Group (ICGG) Gaucher Registry may be obtained at or by calling 1-888-404-4413.

Monitoring Parameters

Neurologic evaluations baseline and repeated every 6 months; adverse effects; weight; platelet count; renal function

Pregnancy Risk Factor C Pregnancy Considerations

Adverse events were observed in animal reproduction studies.

Uncontrolled type 1 Gaucher disease is associated an increased risk of spontaneous abortion; maternal hepatosplenomegaly and thrombocytopenia may also occur and lead to adverse pregnancy outcomes.

Patient Education

What is this drug used for?

• It is used to treat Gaucher's disease.

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

• Dizziness

• Abdominal pain

• Heartburn

• Nausea

• Vomiting

• Passing gas

• Dry mouth

• Back pain

• Constipation

• Leg cramps

• Muscle cramps

• Lack of appetite

• Bloating

• Loss of strength and energy

• Weight loss

• Sensation of heaviness in extremities

• Menstrual changes

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Severe diarrhea

• Burning or numbness feeling

• Vision changes

• Bruising

• Bleeding

• Trouble with memory

• Severe headache

• Tremors

• Abnormal gait

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.

Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.

Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a limited summary of general information about the medicine's uses from the patient education leaflet and is not intended to be comprehensive. This limited summary does NOT include all information available about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not intended to provide medical advice, diagnosis or treatment and does not replace information you receive from the healthcare provider. For a more detailed summary of information about the risks and benefits of using this medicine, please speak with your healthcare provider and review the entire patient education leaflet.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.