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Miglustat (By mouth)

Miglustat (mi-GLOO-stat)

Treats type I Gaucher disease in people who cannot use enzyme replacement therapy.

Brand Name(s):


There may be other brand names for this medicine.

When This Medicine Should Not Be Used:

You should not use this medicine if you have had an allergic reaction to miglustat. Do not use this medicine if you are pregnant.

How to Use This Medicine:


  • Take your medicine as directed.
  • You may take this medicine with or without food.
  • Carefully follow your doctor's instructions about any special diet. This could help control some side effects of this medicine.
  • Swallow the capsule whole. Do not crush, break, or chew it.

If a dose is missed:

  • Take a dose as soon as you remember. If it is almost time for your next dose, wait until then and take a regular dose. Do not take extra medicine to make up for a missed dose.

How to Store and Dispose of This Medicine:

  • Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light.
  • Ask your pharmacist, doctor, or health caregiver about the best way to dispose of any outdated medicine or medicine no longer needed.
  • Keep all medicine out of the reach of children. Never share your medicine with anyone.

Drugs and Foods to Avoid:

Ask your doctor or pharmacist before using any other medicine, including over-the-counter medicines, vitamins, and herbal products.

  • Make sure your doctor knows about all other medicines you are using for Gaucher disease, including Cerezyme® (imiglucerase).
  • If this medicine causes you to have diarrhea, you might need to avoid eating high carbohydrate ("high carb") foods. Talk to your health caregiver about which foods are better for you to eat.

Warnings While Using This Medicine:

  • Using this medicine while you are pregnant can harm your unborn baby. The medicine may also cause birth defects if the father is using it when his sexual partner becomes pregnant. Use an effective method of birth control for the entire time that you are using this medicine. If you are a man, keep using birth control for at least 3 months after your last dose of this medicine. If a pregnancy occurs while you are using this medicine, tell your doctor right away.
  • Make sure your doctor knows if you are breast feeding, or if you have kidney disease.
  • Your doctor will need to check your progress at regular visits while you are using this medicine. Be sure to keep all appointments.
  • Most people who use this medicine have diarrhea (loose, watery stools). You may also throw up or have stomach pain or an upset stomach. You might have gas or constipation (hard, dry bowel movements, less often than usual). Talk to your doctor or health caregiver about how to prevent and treat these problems.

Possible Side Effects While Using This Medicine:

Call your doctor right away if you notice any of these side effects:

  • Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing
  • Hands trembling or shaking, either new or worse than usual.
  • Numbness, tingling, or burning pain in your hands, arms, legs, or feet.
  • Severe diarrhea, nausea, vomiting, weight loss.
  • Trouble seeing.
  • Unusual bleeding or bruising.

If you notice these less serious side effects, talk with your doctor:

  • Headache or dizziness.
  • Mild weight loss.
  • Muscle cramps.

If you notice other side effects that you think are caused by this medicine, tell your doctor

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088

Last Updated: 4/4/2014

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