How does this medication work? What will it do for me?

Miglustat is a glucosylceramide synthase inhibitor. It is used treat mild-to-moderate cases of a rare genetic disease known as type I Gaucher disease, where there is a buildup of glucosylceramide in the body's immune system. Miglustat works by stopping the formation of glucosylceramide. It is used when other therapies for Gaucher disease (such as enzyme replacement therapy) have failed or can't be used.

Miglustat is also used to slow the progression of some of the brain and nervous system symptoms of Niemann-Pick type C disease, a condition where glycosphingolipids (a type of fat) build up in the brain, causing seizures and problems with vision, balance, speech, and memory.

This medication may be available under multiple brand names and/or in several different forms. Any specific brand name of this medication may not be available in all of the forms or approved for all of the conditions discussed here. As well, some forms of this medication may not be used for all of the conditions discussed here.

Your doctor may have suggested this medication for conditions other than those listed in these drug information articles. If you have not discussed this with your doctor or are not sure why you are taking this medication, speak to your doctor. Do not stop taking this medication without consulting your doctor.

Do not give this medication to anyone else, even if they have the same symptoms as you do. It can be harmful for people to take this medication if their doctor has not prescribed it.

What form(s) does this medication come in?

Each white opaque capsule filled with white-to-off-white granulate, contains 100 mg of miglustat. Nonmedicinal ingredients: magnesium stearate, povidone, sodium starch glycolate; capsule shell: gelatin and titanium dioxide

How should I use this medication?

For type I Gaucher disease, the recommended adult dose is 100 mg taken by mouth, 3 times daily at regular intervals.

For Niemann-Pick type C disease, the recommended dose for people over 12 years of age is 200 mg taken by mouth 3 times daily. The children's dose is based on body surface area.

Miglustat should be swallowed whole with a glass of water. It may be taken with or without food.

Many things can affect the dose of medication that a person needs, such as body weight, other medical conditions, and other medications. If your doctor has recommended a dose different from the ones listed here, do not change the way that you are taking the medication without consulting your doctor.

It is important to take this medication exactly as prescribed by your doctor. If you miss a dose, skip the missed dose and continue with your regular dosing schedule. Do not take a double dose to make up for a missed one. If you are not sure what to do after missing a dose, contact your doctor or pharmacist for advice.

Store this medication at room temperature, protect it from moisture, and keep it out of the reach of children.

Do not dispose of medications in wastewater (e.g. down the sink or in the toilet) or in household garbage. Ask your pharmacist how to dispose of medications that are no longer needed or have expired.

Who should NOT take this medication?

Miglustat should not be taken by anyone who:

  • is allergic to miglustat or to any of the ingredients of the medication
  • is pregnant or attempting to become pregnant

What side effects are possible with this medication?

Many medications can cause side effects. A side effect is an unwanted response to a medication when it is taken in normal doses. Side effects can be mild or severe, temporary or permanent. The side effects listed below are not experienced by everyone who takes this medication. If you are concerned about side effects, discuss the risks and benefits of this medication with your doctor.

The following side effects have been reported by at least 1% of people taking this medication. Many of these side effects can be managed, and some may go away on their own over time.

Contact your doctor if you experience these side effects and they are severe or bothersome. Your pharmacist may be able to advise you on managing side effects.

  • constipation
  • decreased appetite
  • diarrhea
  • dizziness
  • dry mouth
  • flu-like symptoms
  • gas
  • generalized weakness
  • headache
  • heartburn
  • muscle cramps
  • nausea
  • sensation of spinning
  • stomach or abdominal pain
  • tremor (shakiness)
  • unusual tiredness
  • vision problems
  • vomiting
  • weight loss

Although most of the side effects listed below don't happen very often, they could lead to serious problems if you do not check with your doctor or seek medical attention.

Check with your doctor as soon as possible if any of the following side effects occur:

  • numbness, burning, or tingling in the hands and feet
  • stomach or abdominal pain (severe)
  • unusual bruising or bleeding

Stop taking the medication and seek immediate medical attention if any of the following occur:

  • seizures

Some people may experience side effects other than those listed. Check with your doctor if you notice any symptom that worries you while you are taking this medication.

Are there any other precautions or warnings for this medication?

Before you begin using a medication, be sure to inform your doctor of any medical conditions or allergies you may have, any medications you are taking, whether you are pregnant or breast-feeding, and any other significant facts about your health. These factors may affect how you should use this medication.

Bleeding: Miglustat may cause a reduced number of platelets in the blood, which can make it difficult to stop cuts from bleeding. If you notice any signs of bleeding, such as frequent nosebleeds, unexplained bruising, or black and tarry stools, notify your doctor as soon as possible.

Diarrhea: Diarrhea is a very common side effect of this medication. If you experience diarrhea, taking miglustat between meals may help. If the diarrhea is bothersome, contact your doctor.

Dizziness/reduced alertness: Miglustat commonly causes dizziness. Avoid driving or operating machinery until you know how this medication affects you.

Kidney disease: Miglustat is removed from the body by the kidneys and its elimination may be reduced in people with kidney disease. If you have kidney disease, discuss with your doctor how this medication may affect your medical condition, how your medical condition may affect the dosing and effectiveness of this medication, and whether any special monitoring is needed.

Severe Gaucher disease: The safety and effectiveness of miglustat have not been evaluated for people with severe Gaucher disease. Miglustat is used for mild-to-moderate type 1 Gaucher disease.

Pregnancy: This medication should not be used by women during pregnancy as it may cause harm to the unborn baby. Both men and women should use a reliable method of contraception. Men should not father a child while they are taking miglustat and for 3 months after stopping the medication. If you become pregnant while you are taking this medication, contact your doctor immediately.

Breast-feeding: It is not known if miglustat passes into breast milk. If you are a breast-feeding mother and are taking this medication, it may affect your baby. Talk to your doctor about whether you should continue breast-feeding.

Children: The safety and effectiveness of using this medication for type I Gaucher disease have not been established for children under 18 years of age. The safety and effectiveness of using this medication for Niemann-Pick type C disease have not been established for children under 4 years of age.

What other drugs could interact with this medication?

Tell your doctor or prescriber about all prescription, over-the-counter (non-prescription), and herbal medications that you are taking. Also tell them about any supplements you take. Since caffeine, alcohol, the nicotine from cigarettes, or street drugs can affect the action of many medications, you should let your prescriber know if you use them. Depending on your specific circumstances, your doctor may want you to:

  • stop taking one of the medications,
  • change one of the medications to another,
  • change how you are taking one or both of the medications, or
  • leave everything as is.

An interaction between two medications does not always mean that you must stop taking one of them. Speak to your doctor about how any drug interactions are being managed or should be managed.

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