Medically reviewed by Drugs.com. Last updated on May 2, 2023.
Uses for miglustat
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.
Miglustat is used in combination with cipaglucosidase alfa-atga injection (Pombiliti™) to treat late-onset of Pompe disease in patients who are not improving on their current enzyme replacement therapy (ERT).
This medicine 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 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.
Appropriate studies on the relationship of age to the effects of Zavesca® capsule 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 this medicine.
Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of Opfolda™ capsule in the elderly.
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 this medicine. Make sure you tell your doctor if you have any other medical problems, especially:
- Diarrhea or
- Heart disease or
- Lung or breathing problems or
- Tremors—Use with caution. May make these conditions worse.
- 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.
Proper use of miglustat
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.
Swallow Opfolda™ capsule whole with unsweetened drinks (eg, water, tea, or coffee with no cream, sugar, or sweeteners). Do not eat or drink for at least 2 hours before and 2 hours after taking this medicine.
Take Opfolda™ capsule 1 hour before receiving Pombiliti™ injection.
Take Zavesca® capsule 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 Pompe disease:
- Adults weighing 50 kilograms (kg) or more—260 milligrams (mg) every other week.
- Adults weighing 40 to less than 50 kg—195 mg every other week.
- Children—Use and dose must be determined by your doctor.
- 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 Pompe 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.
If you miss a dose of Opfolda™ capsule, you should not receive Pombiliti™ injection. If you miss this medicine in combination with Pombiliti™ injection, restart treatment as soon as possible.
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
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.
Using this medicine while you are pregnant can harm your unborn baby. If you are a woman who can get pregnant, your doctor may do tests to make sure you are not pregnant before receiving this medicine. Use an effective form of birth control during treatment and for at least 60 days after the last dose. If you think you have become pregnant while using this medicine, tell your doctor right away.
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.
Some men and women who use this medicine have become infertile (unable to have children). Talk with your doctor before using this medicine if you plan to have children.
Side Effects of miglustat
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:
- Black, tarry stools
- bleeding gums
- blood in the urine or stools
- burning, crawling, itching, numbness, prickling, "pins and needles", or tingling feelings
- pinpoint red spots on the skin
- trembling or shaking in the hands
- unsteadiness or awkwardness
- unusual bleeding or bruising
- weakness in the arms, hands, legs, or feet
- weight loss
- Chest tightness
- fast, pounding, or irregular heartbeat or pulse
- increase blood pressure
- lower back or side pain
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:
- Back pain
- bloated or full feeling
- blurred or loss of vision
- difficulty having a bowel movement (stool)
- disturbed color perception
- double vision
- dry mouth
- excess air or gas in the stomach or intestines
- halos around lights
- heaviness in the limbs
- leg cramps
- loss of appetite
- memory loss
- menstrual changes
- pain or discomfort in the chest, upper stomach, or throat
- passing gas
- stomach discomfort, upset or pain
- swelling of the abdominal or stomach area
- tunnel vision
- unsteady walk
- Change in taste
- joint or muscle pain
- lack or loss of strength
- loss of taste
- pressure in the stomach
- stomach discomfort, upset, or pain
- swelling of the stomach area
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.
Commonly used brand name(s)
In the U.S.
Available Dosage Forms:
Therapeutic Class: Endocrine-Metabolic Agent
Pharmacologic Class: Glucosylceramide Synthase Inhibitor
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