Frequently asked questions (FAQs) about ZAVESCA^® (miglustat)

Currently, there are 2 ways to treat type 1 Gaucher disease. One way is to decrease the body's over-accumulation of the harmful fatty substances by limiting production of glycosphingolipids (GSL). The other way is enzyme replacement therapy (ERT), where man-made enzyme is added to the body to help break down the fatty substances. ZAVESCA is also different because, as a small molecule, it goes throughout the body.

Yes. Studies with ZAVESCA included both people who had previously taken Cerezyme (imiglucerase)—also called enzyme replacement therapy—but had stopped taking ERT for at least 6 months, and people who had never been treated with ERT.¹

Within 6 months, people treated with ZAVESCA had improvements in 4 key areas¹:

• Reduction in the size of livers
• Reduction in size of spleens
• Platelet counts increased
• Hemoglobin concentration increased

Your doctor will generally monitor these 4 key areas in addition to other tests to see how your treatment is doing.

It's important to take ZAVESCA as prescribed by your doctor. The recommended dose of ZAVESCA is usually 1 pill (100 mg) taken 3 times each day. In some cases, your doctor may have you take more or less, depending on what he or she thinks is right for you.

You should take ZAVESCA at the same time each day. For example, if your prescription is for ZAVESCA 3 times a day, then take it when you first wake up, in the early afternoon, and before going to bed (or as close to 8-hour intervals as possible). Swallow ZAVESCA capsule whole with water.¹

You can take ZAVESCA with or without food. Some people experience fewer side effects when taking ZAVESCA without food, while others see the opposite effect. You can discuss with your doctor what works best for you.¹

You should NOT take ZAVESCA if you are pregnant or nursing because ZAVESCA can harm a fetus. Do not use ZAVESCA if you plan to become pregnant, or if your partner can become pregnant. You must use reliable birth control while you are taking ZAVESCA.¹

ZAVESCA may cause changes in sperm development and function. If you are a man, you should use birth control while you are taking ZAVESCA and you should not father a child for at least 3 months after you stop taking ZAVESCA.¹

It's important to talk to your doctor if any of the following apply to you¹:

• If you are breast-feeding. It is not known if ZAVESCA passes into your milk, or if it can harm your baby. You should decide either to breast feed or take ZAVESCA, but not both.
• If you have kidney problems. There are special recommendations for taking ZAVESCA, so your doctor may prescribe a different dose if you have kidney problems.
• If you have existing neuropathy or a disease that causes tremor, like Parkinson's disease.
• If you are lactose intolerant or have other forms of chronic diarrhea.

ZAVESCA has not been studied in patients with type 1 Gaucher disease under 18 years of age.

ZAVESCA is not broken down by the liver, where some drug interactions occur. However, ZAVESCA has not been studied with all drugs.

Talk to your doctor or pharmacist before taking any other drugs.

ZAVESCA may cause serious side effects including:

• Problems affecting your nerves (neurologic problems):

  New or worse hand tremors (shaky movements). Tremors may begin within the first month of starting treatment. Sometimes the tremors may go away between 1 to 3 months with continued treatment. Sometimes a lower dose or stopping ZAVESCA is needed to help the tremors go away. Call your doctor if you get hand tremors while taking ZAVESCA or the hand tremors you already have get worse.

  Numbness and tingling in your hands, arms, legs, or feet (peripheral neuropathy). Call your doctor right away if you get numbness or tingling in your arms or legs.

  Your doctor may test your nerves (neurological exam) before you start ZAVESCA and may repeat this procedure at a later time.

• Diarrhea. Your doctor may give you another medicine (anti-diarrheal) to treat diarrhea if it is a problem for you, and may recommend changes to your diet.
• Weight loss. You may also lose weight when you start treatment with ZAVESCA.
• Low Platelet Count. Your doctor may do blood tests to monitor your blood platelet count.

The most common side effects of ZAVESCA are:

Call your doctor if you have any side effect that bothers you or that does not go away. These are not all the side effects with ZAVESCA. For more information, ask your doctor or your pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA 1088.

Please see full Prescribing Information.