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.

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.

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

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.

You may need blood tests.

Store at room temperature away from moisture and heat.

Usual Adult Dose for Gaucher Disease:

Usual dose: 100 mg orally 3 times a day at regular intervals

Comments:
-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)

You should not use miglustat if you are allergic to it.

Tell your doctor if you have ever had:

• stomach problems;

• an intestinal disorder such as ulcerative colitis;

• kidney disease; or

• a condition that causes tremors.

Miglustat may harm an unborn baby. Use effective birth control to prevent pregnancy, and tell your doctor if you become pregnant.

You should not breastfeed while using miglustat.

Do not give this medication to anyone under 18 years old without medical advice.

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

Get your prescription refilled before you run out of medicine completely.

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

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

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.

Miglustat can cause diarrhea, which may be serious. Your doctor may prescribe anti-diarrhea medicine and may also recommend changes in your diet.

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

Miglustat may cause serious side effects. 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 of miglustat may include:

• gas, constipation, bloating;

• nausea, vomiting, stomach pain, indigestion, loss of appetite, weight loss;

• dry mouth;

• dizziness, weakness;

• headache (including migraine);

• back pain, muscle cramps, leg cramps;

• pain or heavy feeling in your arms or legs;

• memory problems, feeling unsteady, loss of balance;

• burning pain in your hands or feet

• 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.

Other drugs may affect miglustat, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

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.