miglustat (Oral route)
Commonly used brand name(s)
In the U.S.
Available Dosage Forms:
Therapeutic Class: Endocrine-Metabolic Agent
Pharmacologic Class: Glucosylceramide Synthase Inhibitor
Uses For miglustat
Miglustat is used to treat adults with mild to moderate type 1 Gaucher disease. Miglustat is only used in people who cannot be treated with enzyme replacement therapy. Type 1 Gaucher disease is a disease you get from both your parents. People with type 1 Gaucher disease are missing an enzyme (naturally occurring substance in your body) that breaks down a chemical in your body called glucosylceramide. Too much glucosylceramide causes liver and spleen enlargement, changes in the blood, and bone disease. Miglustat works by stopping the body from making glucosylceramide.
miglustat is available only with your doctor's prescription.
Before Using miglustat
In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For miglustat, the following should be considered:
Tell your doctor if you have ever had any unusual or allergic reaction to miglustat or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.
miglustat is not used in children under 18 years of age.
Many medicines have not been specifically studied in older people. Therefore, it may not be known whether they work the same way the do in younger adults. miglustat is not expected to cause different side effects or problems in older people than it does in younger adults.
|All Trimesters||X||Studies in animals or pregnant women have demonstrated positive evidence of fetal abnormalities. This drug should not be used in women who are or may become pregnant because the risk clearly outweighs any possible benefit.|
There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.
Interactions with Medicines
Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.
Interactions with Food/Tobacco/Alcohol
Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.
Other Medical Problems
The presence of other medical problems may affect the use of miglustat. Make sure you tell your doctor if you have any other medical problems, especially:
- Gaucher disease, Type 1, severe—miglustat is not currently being used in patients with severe Type 1 Gaucher disease
- Kidney disease—this condition may cause you to have more miglustat in your body; your doctor may want to change the amount of miglustat that you take
Proper Use of miglustat
It is important to take miglustat exactly as your doctor prescribed. You should take your medicine at the same time or at the same times each day.
The capsules should be swallowed whole with water and may be taken with or without food. Check with your doctor or pharmacist if you have any questions.
Your doctor may recommend changes to your diet to help with some side effects. It is important that you follow these changes.
The dose of miglustat will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of miglustat. If your dose is different, do not change it unless your doctor tells you to do so.
The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.
- For oral dosage form (capsules):
- For Mild to Moderate Type 1 Gaucher disease
- Adults—One 100 milligram (mg) capsule given three times a day; your doctor may change this dose as needed
- Children—Use is not recommended in children under the age of 18
- For Mild to Moderate Type 1 Gaucher disease
If you miss a dose of miglustat, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.
Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.
Keep out of the reach of children.
Do not keep outdated medicine or medicine no longer needed.
Ask your healthcare professional how you should dispose of any medicine you do not use.
Precautions While Using miglustat
Miglustat can cause problems affecting your nerves. If you have hand tremors (shaky movements) or if miglustat worsens a hand tremor you already have call your doctor. Your doctor might want to change your dose of miglustat.
If you experience numbness and tingling in your hands, arms, legs, or feet (peripheral neuropathy) call your doctor right away.
It is very important that your doctor check you at regular visits. Your doctor will also want to test your nerves (neurological exam) before you start taking miglustat and may repeat this test at a later time.
Diarrhea is the most common side effect for people taking miglustat. Your doctor may give you another medicine (anti-diarrheal) to help treat diarrhea if it is a problem for you. Your doctor may also recommend changes to your diet. You may also lose weight when you start treatment with miglustat.
It is very important to discuss with your doctor if you are pregnant or plan to become pregnant before starting miglustat. You should use effective birth control while taking miglustat. Miglustat may also harm a man's sperm. All men should use effective birth control during treatment and for three months after stopping treatment.
miglustat Side Effects
Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.
Check with your doctor immediately if any of the following side effects occur:More common
- Black, tarry stools
- bleeding gums
- blood in urine or stools
- burning, crawling, itching, numbness, prickling, "pins and needles", or tingling feelings
- pinpoint red spots on skin
- unusual bleeding or bruising
- Painful sensations
- shakiness in legs, arms, hands, feet
- trembling or shaking of hands or feet
- unsteadiness or awkwardness
- weakness in arms, hands, legs, or feet
Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:More Common
- Acid or sour stomach
- back pain
- bloated, full feeling
- change in vision
- difficulty having a bowel movement (stool)
- dry mouth
- excess air or gas in stomach or intestines
- full or bloated feeling or pressure in the stomach
- heaviness in limbs
- leg cramps
- loss of appetite
- memory loss
- menstrual changes
- pain or discomfort in chest, upper stomach, or throat
- passing gas
- stomach discomfort, upset or pain
- swelling of abdominal or stomach area
- unsteady walk
- weight loss
Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
See also: miglustat side effects (in more detail)
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