Commonly Used Brand Name(s)Zavesca
Therapeutic ClassificationsEndocrine-Metabolic Agent
Pharmacologic ClassificationsGlucosylceramide Synthase Inhibitor
Miglustat is used to treat mild to moderate type 1 Gaucher disease in people who cannot be treated with an enzyme replacement therapy. Type 1 Gaucher disease is a disease you get from both your parents. People with type 1 Gaucher disease lack 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.
This medicine is available only with your doctor's prescription.
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Take this medicine exactly as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered. To do so may increase the chance of side effects.
This medicine comes with a patient information leaflet. Read and follow these instructions carefully. Ask your doctor if you have any questions.
Take this medicine at the same time each day.
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 this medicine 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 this medicine. 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 type 1 Gaucher's disease:
- Adults—100 milligrams (mg) three times a day. Your doctor may adjust your dose as needed.
- Children—Use and dose must be determined by your doctor.
- For type 1 Gaucher's disease:
If you miss a dose of this medicine, skip the missed dose and go back to your regular dosing schedule. Do not double doses.
Use & StorageTOP
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Do not keep outdated medicine or medicine no longer needed.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.
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 this medicine, the following should be considered:
Tell your doctor if you have ever had any unusual or allergic reaction to this medicine 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.
Appropriate studies have not been performed on the relationship of age to the effects of miglustat in the pediatric population. Safety and efficacy have not been established.
Although appropriate studies on the relationship of age to the effects of miglustat have not been performed in the geriatric population, no geriatric-specific problems have been documented to date. However, elderly patients are more likely to have age-related liver, kidney, or heart problems, which may require caution and an adjustment in the dose for patients receiving miglustat.
|All Trimesters||C||Animal studies have shown an adverse effect and there are no adequate studies in pregnant women OR no animal studies have been conducted and there are no adequate studies in pregnant women.|
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.
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.
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 ProblemsTOP
The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:
- Kidney disease, mild or moderate—Use with caution. The effects may be increased because of slower removal of the medicine from the body.
- Kidney disease, severe—Use is not recommended in patients with this condition.
It is very important that your doctor check your progress at regular visits to make sure that this medicine is working properly. Blood and nerve tests (neurological exam) before and during treatment may be needed to check for unwanted effects.
Check with your doctor right away if you are having burning, numbness, tingling, or painful sensations in the arms, hands, legs, or feet. These could be symptoms of a condition called peripheral neuropathy.
Taking this medicine may decrease in the number of platelets in the blood. Your doctor may monitor this periodically during therapy.
This medicine may cause or worsen hand tremors (trembling or shaking of the hands). Tell your doctor right away if you develop new hand tremors or worsening of existing hand tremors while using this medicine.
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.