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Why is this medication prescribed?
Miglustat is used to treat Gaucher disease type 1 (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 is in a class 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.
How should this medicine be used?
Miglustat comes as a capsule to take by mouth. It is usually taken with or without food and with plenty of water up to three times a day. To help you remember to take miglustat, take it at around the same time(s) every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take miglustat exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.
Swallow the capsules whole; do not split, chew, or crush them.
Miglustat controls Gaucher disease but does not cure it. Continue to take miglustat even if you feel well. Do not stop taking miglustat without talking to your doctor.
Other uses for this medicine
This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.
What special precautions should I follow?
Before taking miglustat,
- Tell your doctor and pharmacist if you are allergic to miglustat, any other medications, or soy products.
- Tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking. Be sure to mention imiglucerase (Cerezyme). Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
- Tell your doctor if you have or have ever had a tremor (shaking of your hands that you cannot control); pain, burning, tingling, or numbness in your hands or feet; any disease that affects your nervous system; or kidney disease.
- Tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. You should use effective birth control to prevent pregnancy while you are taking miglustat. If you become pregnant while taking miglustat, call your doctor immediately. Miglustat may harm the fetus.
- You should know that miglustat may damage sperm. Men who are taking miglustat should use effective birth control during treatment and for 3 months afterward.
What special dietary instructions should I follow?
Miglustat may cause diarrhea and weight loss. If you experience these side effects, your doctor will tell you how to change your diet to improve your symptoms. You may be told to avoid foods that are high in carbohydrates. Follow these instructions carefully.
What should I do if I forget a dose?
Skip the missed dose and continue your regular dosing schedule. Do not take a double dose to make up for a missed one.
What side effects can this medication cause?
Miglustat may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:
- Stomach pain or bloating
- Loss of appetite
- Weight loss
- Upset stomach
- Dry mouth
- Muscle cramps, especially in the legs
- Feeling of heaviness in the arms or legs
- Unsteadiness when walking
- Back pain
- Memory problems
- Difficult or irregular menstruation (period)
Some side effects can be serious. The following symptoms are uncommon, but if you experience any of them, call your doctor immediately:
- 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 may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.
If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration’s (FDA) MedWatch Adverse Event Reporting program online (http://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).
What should I know about storage and disposal of this medication?
Keep this medication in the container it came in, tightly closed, and out of reach of children. Store it at room temperature and away from excess heat and moisture (not in the bathroom).
Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA’s Safe Disposal of Medicines website (http://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.
It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location – one that is up and away and out of their sight and reach. http://www.upandaway.org
In case of emergency/overdose
In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can’t be awakened, immediately call emergency services at 911.
Symptoms of overdose may include:
- Pain, burning, tingling , or numbness in your hands, arms, legs, or feet
- Sore throat, fever, chills, or other signs of infection
What other information should I know?
Keep all appointments with your doctor.
Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.
It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.
Last Revised – 08/15/2018