Frequently Asked Questions (FAQ) about Zavesca

In people with type 1 Gaucher disease, the body has an excess of certain types of fatty substances called glycosphingolipids (gli-coh-sfing-goh-lip-ids), also called GSL.¹ Normally, the body produces GSL and certain enzymes break it down. In people with type 1 Gaucher disease there's either not enough of the enzyme or it doesn't work properly, so the fatty substances build up in the body's cells over time—causing serious problems in parts of the body like the liver, spleen, lungs, and bones.¹

Zavesca reduces the harmful buildup of the fatty substances by reducing the amount of GSL that the body produces. This way the enzymes you to have are able break down the fatty substances, so that it decreases the harmful accumulation. Zavesca also helps increase the ability of the natural enzyme in your body to work more effectively to remove the GSLs.

Currently, there are two 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. The other way is enzyme replacement therapy (ERT), where man-made enzyme is added to the body to help break down the fatty substances.

Yes! Zavesca has been studied in adult patients with mild to moderate type 1 Gaucher disease. The studies with Zavesca included both people who had previously taken Cerezyme (imiglucerase)—also called Enzyme Replacement Therapy (ERT)—but had stopped taking ERT for at least 6 months, and in people who had never been previously treated.¹

Within as little as 6 months, people treated with Zavesca had improvements in 4 key areas:

• There was a reduction in the size of livers and spleens.¹
• Hemoglobin concentration and platelet counts increased.¹

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

Yes! Zavesca has been studied in adult patients with mild to moderate type 1 Gaucher disease who had never previously been treated because ERT was not a therapeutic option.¹

Within as little as 6 months, people treated with Zavesca had improvements in 4 key areas:

• There was a reduction in the size of livers and spleens.¹
• Hemoglobin concentration and platelet counts increased.¹

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 usual regiment is 1 pill (100mg) taken 3 times each day. In some cases, your doctor may adjust that dose depending on what he (or she) thinks is right for you.¹

You should take Zavesca at the same time each day. For instance, 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

If you miss a dose of Zavesca, simply skip that dose. Take the next Zavesca capsule at the usual time.¹

No blood tests are required to take Zavesca. However, your doctor may want to monitor your type 1 Gaucher disease activity by taking blood samples regularly to determine your levels of hemoglobin, platelet counts, and important disease markers. Your doctor will let you know how often to have these blood tests. Your doctor may also measure the size of your liver and spleen with physical examinations and radiological tests.

There is currently no cure for Type 1 Gaucher Disease. You can stay on Zavesca as long as your doctor wants you to.

Yes, diarrhea and gas are two of the most common side effects for people taking Zavesca.¹

The way Zavesca works is by limiting overproduction of fatty substances in your body's cells called glycosphingolipids (gli-coh-sfing-goh-lip-ids), or GSLs.¹ The same way Zavesca decreases production of the GSLs, it also limits how the body digests carbohydrates.¹

Carbohydrates are found in grains, bread, rice, and flour. Simple sugars, found in things like soda, cookies, or desserts, are also carbohydrates. Zavesca may affect how long carbohydrates stay in the intestines, so carbohydrates may be digested more slowly than normal and can cause diarrhea and gas. While many people who take Zavesca may get gas, loose stools, or diarrhea, in most patients these side effects will go away or decrease over time.¹

Taking the simple step of reducing carbohydrates in your diet may help manage these side effects. Your doctor may also recommend over-the-counter medications to help control gas and diarrhea.

Yes. Some patients have reported numbness and tingling in the hands, arms, legs, or feet (peripheral neuropathy). You should 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 at a later time.¹

If you develop hand tremors (shaky movements like you've had too much coffee) or existing tremors get worse, you should call your doctor. They may lower the dose or stop Zavesca to help manage the tremors.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

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 you:¹

• If you are pregnant or planning to become pregnant. You should not take Zavesca.
• 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.
• Zavesca has not been studied in patients with type 1 Gaucher disease under 18 years of age.
• 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 does not affect liver metabolism which is responsible for most drug interactions, so it is unlikely that Zavesca will interact with other drugs.

Zavesca may decrease the levels of Cerezyme but the data are inconclusive. Talk to your doctor or pharmacist if you're taking any other medications.