Generic Name: miglustat (mi GLOO stat)
Brand Name: Zavesca
What is miglustat?
Miglustat reduces the formation of a certain protein in the body in people with type 1 Gaucher disease.
Gaucher disease is a genetic condition in which the body lacks the enzyme needed to break down certain fatty materials (lipids). Lipids can build up in the body, causing symptoms such as easy bruising or bleeding, weakness, anemia, bone or joint pain, enlarged liver or spleen, or weakened bones that are easily fractured.
Miglustat is used to treat mild to moderate type 1 Gaucher disease by people who cannot receive enzyme replacement therapy.
Miglustat may improve the condition of the liver, spleen, bones, and blood cells in people with Type I Gaucher disease. However, miglustat is not a cure for this condition.
Miglustat may also be used for purposes not listed in this medication guide.
What is the most important information I should know about miglustat?
Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.
What should I discuss with my health care provider before taking miglustat?
You should not use miglustat if you are allergic to it.
To make sure miglustat is safe for you, tell your doctor if you have:
an intestinal disorder such as ulcerative colitis;
kidney disease; or
a condition that causes tremors.
FDA pregnancy category C. It is not known whether miglustat will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant while using this medicine.
It is not known whether miglustat passes into breast milk or if it could harm a nursing baby. You should not breast-feed while using this medicine.
Do not give this medication to anyone under 18 years old without medical advice.
How should I take miglustat?
Follow all directions on your prescription label. Your doctor may occasionally change your dose to make sure you get the best results. Do not take this medicine in larger or smaller amounts or for longer than recommended.
Miglustat is usually taken 3 times per day. Take the medicine at the same time each day.
Your doctor will need to check your nerve and muscle function every 6 months while you are using miglustat.
Use miglustat regularly to get the most benefit. Get your prescription refilled before you run out of medicine completely.
Store at room temperature away from moisture and heat.
What happens if I miss a dose?
If you miss a dose of miglustat, skip the missed dose and only take the next regularly scheduled dose. Do not take a double dose of this medication unless otherwise directed by your doctor.
What happens if I overdose?
Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.
What should I avoid while taking miglustat?
Miglustat can cause diarrhea, which may be serious. Your doctor may prescribe anti-diarrhea medicine and may also recommend changes in your diet.
Follow your doctor's instructions about any restrictions on food, beverages, or activity.
Miglustat side effects
Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.
Call your doctor at once if you have:
tremor or uncontrolled shaking, especially in your hands;
numbness, tingling, or burning pain in your hands or feet;
severe diarrhea and weight loss; or
easy bruising or bleeding (nosebleeds, bleeding gums).
Common side effects may include:
diarrhea, constipation, bloating;
nausea, vomiting, stomach pain, indigestion;
headache (including migraine);
back pain, muscle cramps;
pain or heavy feeling in your arms or legs;
memory problems, feeling unsteady, loss of balance;
vision problems; or
changes in your menstrual periods.
This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
See also: Side effects (in more detail)
Miglustat dosing information
Usual Adult Dose for Gaucher Disease:
Initial dose: 100 mg orally 3 times a day at regular intervals
Approved indication: As monotherapy for the treatment of mild to moderate type 1 Gaucher disease when enzyme replacement therapy is not a therapeutic option (e.g., due to allergy, hypersensitivity, or poor venous access)
What other drugs will affect miglustat?
Other drugs may interact with miglustat, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell each of your health care providers about all medicines you use now and any medicine you start or stop using.
More about miglustat
- Other brands: Zavesca
Related treatment guides
Where can I get more information?
- Your pharmacist can provide more information about miglustat.
- Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.
- Disclaimer: Every effort has been made to ensure that the information provided by Cerner Multum, Inc. ('Multum') is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Multum information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Multum does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Multum's drug information does not endorse drugs, diagnose patients or recommend therapy. Multum's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners. The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Multum does not assume any responsibility for any aspect of healthcare administered with the aid of information Multum provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are taking, check with your doctor, nurse or pharmacist.
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