Miglustat

Miglustat is used to treat mild to moderate type 1 Gaucher disease in adults who cannot have enzyme replacement therapy.

Miglustat may also be used for other reasons that are not mentioned in this medication guide.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may sometimes change your dose. Use the medicine exactly as directed.

Miglustat is usually taken 3 times per day at the same time each day.

You may be given other medications to help treat certain side effects. Keep taking these medicines for as long as your doctor has prescribed.

You may need to follow a special diet that avoids foods high in carbohydrates. Follow all instructions from your doctor or dietitian. Learn about the foods you should eat or avoid.

You may need medical tests to check your nerve function before and during treatment with miglustat.

Your blood may also need to be tested while you are using miglustat.

Store at room temperature away from moisture and heat.

Use only as directed. Inform your doctor if you are taking other medicines, have other medical conditions, or have allergies.

Tell your doctor if you have or have ever had:

• inflammatory bowel disease;
• nervous system problems;
• a condition that causes tremors or shaking; or
• kidney disease.

Miglustat may harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

Do not breastfeed while using miglustat.

Get emergency medical help if you have signs of an allergic reaction: hives, difficulty breathing, swelling of your face, lips, tongue, or throat.

Miglustat may cause serious side effects. Call your doctor right away 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
• low levels of platelets in your blood--easy bruising or bleeding (nosebleeds, bleeding gums).

Common side effects of miglustat may include:

• gas, bloating, constipation;
• nausea, vomiting, loss of appetite, weight loss, stomach pain or upset;
• dry mouth;
• dizziness, weakness;
• headache (including migraine);
• back pain, muscle cramps, leg cramps;
• heavy feeling in your arms or legs;
• memory problems, loss of balance or coordination;
• numbness, tingling, or burning pain in your skin;
• 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.

Usual Adult Dose for Gaucher Disease:

Usual dose: 100 mg orally 3 times a day at regular intervalsComments: -Decreasing consumption of foods high in disaccharides (e.g., lactose or sucrose) and not taking this drug with food has been shown to reduce the risk and/or intensity of gastrointestinal adverse events. -Dose reduction to 100 mg orally once or twice a day may be necessary due to adverse reactions such as tremor or diarrhea.Use: 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)

Skip the missed dose and take your next dose at the usual time. Do not take two doses at once.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Other drugs may impact miglustat, such as prescription and over-the-counter medicines, vitamins, and herbal products. Inform your doctor about all other medicines you are using.