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Generic name: miglustat [ mi-GLOO-stat ]
Brand name: Zavesca
Dosage form: oral capsule (100 mg)
Drug class: Miscellaneous metabolic agents

Medically reviewed by on Apr 14, 2021. Written by Cerner Multum.

What is miglustat?

Gaucher disease is a genetic condition in which the body lacks the enzyme needed to break down certain fatty materials (lipids). Lipids can build up in the body, causing symptoms such as easy bruising or bleeding, weakness, anemia, bone or joint pain, enlarged liver or spleen, or weakened bones that are easily fractured.

Miglustat is used to treat mild to moderate type 1 Gaucher disease by people who cannot receive enzyme replacement therapy.

Miglustat may improve the condition of the liver, spleen, bones, and blood cells in people with Type I Gaucher disease. However, miglustat is not a cure for this condition.

Miglustat may also be used for purposes not listed in this medication guide.


Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

Before taking this medicine

You should not use miglustat if you are allergic to it.

Tell your doctor if you have ever had:

  • stomach problems;

  • an intestinal disorder such as ulcerative colitis;

  • kidney disease; or

  • a condition that causes tremors.

Miglustat may harm an unborn baby. Use effective birth control to prevent pregnancy, and tell your doctor if you become pregnant.

You should not breastfeed while using miglustat.

Do not give this medication to anyone under 18 years old without medical advice.

How should I take miglustat?

Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose. Use the medicine exactly as directed.

Miglustat is usually taken 3 times per day. Take the medicine at the same time each day.

Your doctor will need to check your nerve and muscle function every 6 months while you are using miglustat.

You may need blood tests.

Store at room temperature away from moisture and heat.

What happens if I miss a dose?

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

Get your prescription refilled before you run out of medicine completely.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while taking miglustat?

Miglustat can cause diarrhea, which may be serious. Your doctor may prescribe anti-diarrhea medicine and may also recommend changes in your diet.

Miglustat side effects

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Miglustat may cause serious side effects. Call your doctor at once if you have:

  • tremor or uncontrolled shaking, especially in your hands;

  • numbness, tingling, or burning pain in your hands or feet;

  • severe diarrhea and weight loss; or

  • easy bruising or bleeding (nosebleeds, bleeding gums).

Common side effects of miglustat may include:

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Miglustat dosing information

Usual Adult Dose for Gaucher Disease:

Usual dose: 100 mg orally 3 times a day at regular intervals

-Decreasing consumption of foods high in disaccharides (e.g., lactose or sucrose) and not taking this drug with food has been shown to reduce the risk and/or intensity of gastrointestinal adverse events.
-Dose reduction to 100 mg orally once or twice a day may be necessary due to adverse reactions such as tremor or diarrhea.

Use: As monotherapy for the treatment of mild to moderate type 1 Gaucher disease when enzyme replacement therapy is not a therapeutic option (e.g., due to allergy, hypersensitivity, or poor venous access)

What other drugs will affect miglustat?

Other drugs may affect miglustat, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

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