Miglustat

Miglustat is used to treat an enzyme deficiency known as type 1 Gaucher disease. It is only for people who cannot tolerate enzyme replacement therapy.

Miglustat Overview

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Miglustat is a prescription medication used to treat type 1 Gaucher's disease – a condition in which a certain fatty substance is not broken down normally in the body and instead builds up in some organs and causes liver, spleen, bone, and blood problems.

Miglustat belongs to a group of medications called enzyme inhibitors. It works by preventing the body from producing the fatty substance so that less of it will build up in the body and cause symptoms.

This medication comes in capsule form and is usually taken 3 times a day.

Do not chew, divide, or break miglustat capsules. Swallow the capsules whole.

Common side effects of miglustate include diarrhea, weight loss, stomach pain, gas, nausea and vomiting, headache including migraine, tremor, leg cramps, weakness, vision problems, thrombocytopenia, muscle cramps, back pain, constipation, dry mouth, heaviness in arms and legs, memory loss, unsteady walking, anorexia, indigestion, paresthesia, stomach bloating, stomach pain not related to food, and menstrual changes.

Miglustat can also cause dizziness. Do not drive or operate heavy machinery until you know how miglustat affects you.

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Miglustat Cautionary Labels

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Uses of Miglustat

Miglustat is a prescription medication used to treat type 1 Gaucher's disease – a condition in which a certain fatty substance is not broken down normally in the body and instead builds up in some organs and causes liver, spleen, bone, and blood problems.

This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.

Miglustat Brand Names

Miglustat may be found in some form under the following brand names:

Miglustat Drug Class

Miglustat is part of the drug class:

Side Effects of Miglustat

Serious side effects have been reported with miglustat. See the “Miglustat Precautions” section.

Common side effects of miglustat include the following:

  • diarrhea
  • stomach pain or bloating
  • gas
  • loss of appetite
  • weight loss
  • upset stomach
  • vomiting
  • constipation
  • indigestion
  • dry mouth
  • weakness
  • muscle cramps, especially in the legs
  • feeling of heaviness in the arms or legs
  • unsteadiness when walking
  • back pain
  • dizziness
  • nervousness
  • headache
  • memory problems
  • difficult or irregular menstrual periods

This is not a complete list of miglustat side effects. Ask your doctor or pharmacist for more information.

Tell your doctor if you have any side effect that bothers you or that does not go away.

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

Miglustat Interactions

Tell your doctor about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements. Especially tell your doctor if you take imiglucerase (Cerezyme).

This is not a complete list of miglustat drug interactions. Ask your doctor or pharmacist for more information.

Miglustat Precautions

Serious side effects have been reported with miglustat including the following:

  • pain, burning, numbness or tingling in your hands, arms, legs, or feet
  • shaking hands that you cannot control
  • changes in vision
  • easy bruising or bleeding

MIglustat can cause dizziness. Do not drive or operate heavy machinery until you know how miglustat affects you.

Do not take miglustat if you are allergic to miglustat or to any of its ingredients.

Miglustat Food Interactions

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case of miglustat, there are no specific foods that you must exclude from your diet when receiving this medication.

Inform MD

Before taking miglustsat, tell your doctor about all of your medical conditions. Especially tell your doctor if you:

  • are allergic to miglustat or to any of its ingredients
  • have or have ever had a tremor (shaking of your hands that you cannot control
  • have or have had pain, burning, tingling, or numbness in your hands or feet
  • have or have had any disease that affects your nervous system
  • have or have had kidney disease
  • are pregnant or breastfeeding

Tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements.

Miglustat and Pregnancy

Tell your doctor if you are pregnant or plan to become pregnant.

The FDA categorizes medications based on safety for use during pregnancy. Five categories - A, B, C, D, and X – are used to classify the possible risks to an unborn baby when a medication is taken during pregnancy.

Miglustat falls into category C. In animal studies, pregnant animals were given this medication and had some babies born with problems. No well-controlled studies have been done in humans. Therefore, this medication may be used if the potential benefits to the mother outweigh the potential risks to the unborn child.

Miglustat and Lactation

Tell your doctor if you are breastfeeding or plan to breastfeed.

It is not known if miglustat crosses into human milk. Because many medications can cross into human milk and because of the possibility for serious adverse reactions in nursing infants with use of this medication, a choice should be made whether to stop nursing or stop the use of this medication. Your doctor and you will decide if the benefits outweigh the risk of using miglustat.

Miglustat Usage

Take miglustat exactly as prescribed.

This medication comes in capsule form and is usually taken 3 times a day.

Do not chew, divide, or break miglustat capsules. Swallow the capsules whole.

Your doctor may prescribe another medicine (anti-diarrheal) to treat diarrhea if it is a problem for you and may recommend changes to your diet, such as avoiding foods high in carbohydrates while you are taking miglustat. Talk with your doctor about your diet if you have diarrhea.

If you miss a dose, take the missed dose as soon as you remember. If it is almost time for the next dose, skip the missed dose and take your next dose at the regular time. Do not take two doses of miglustat at the same time.

Miglustat Dosage

Take this medication exactly as prescribed by your doctor. Follow the directions on your prescription label carefully.

The dose your doctor recommends may be based on the following:

  • the condition being treated
  • other medical conditions you have
  • how you respond to this medication

The recommended dose of miglustat is 100 mg three times daily. The dose may be reduced in patients who experience side effects or who have kidney problems.

Miglustat Overdose

If you take too much miglustat, call your healthcare provider or local Poison Control Center, or seek emergency medical attention right away.

If miglustat is administered by a healthcare provider in a medical setting, it is unlikely that an overdose will occur. However, if overdose is suspected, seek emergency medical attention.

Forms of Medication


Other Requirements

  • Store miglustat at room temperature.
  • Protect from excess heat and moisture.
  • Keep this and all medicines out of the reach of children.