Zavesca®

What is Zavesca® (miglustat)?
Zavesca® is a prescription medicine take by mouth for adults with mild to moderate type 1 Gaucher disease. Zavesca® is used only in patients who cannot be treated with enzyme replacement therapy.

How does Zavesca® work?
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) or GSL. Normally, the body produces GSL and a certain enzyme breaks 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 over time, causing serious problems in parts of the body like the liver, spleen, lungs, and bones. Zavesca® reduces the harmful buildup of fatty substances throughout the body by reducing the amount of GSL the body produces. Zavesca® also helps increase the ability of natural enzymes in the body to work more effectively in its cells. Zavesca® helps treat the symptoms of type 1 Gaucher disease because the enzymes you do have are able to break down the fatty substances and reduce the buildup.

Zavesca® has been shown to provide important benefits for people with type 1 Gaucher disease, whether they have been treated before with enzyme replacement therapy (ERT) or they are being treated for the first time.1 Individual results may vary.

Zavesca®:
* Reduces swelling of the liver and spleen
* Increases amounts of hemoglobin and platelets

Taking Zavesca®
It's important to take Zavesca® as prescribed by your doctor. Zavesca® is the only oral medication approved for type 1 Gaucher disease. The recommended dose of Zavesca® is usually 1 pill (100 mg) 3 times a day, but your doctor will tell you which dose is right for you.

Giving it time
Zavesca® may help keep your disease under control and make a positive difference in your life. Remember, different people react to Zavesca® in different ways.

Like many drugs, Zavesca® may cause side effects. Many of these side effects, if they occur, may lessen or go away over time. Still, you should be aware of side effects that may occur while you are taking Zavesca®.

Diarrhea is the most common side effect for people taking Zavesca®. Your doctor may give you a medicine (anti-diarrheal) to manage diarrhea if it is a problem for you, and may recommend dietary changes to reduce high-carbohydrate foods at the start of your treatment with Zavesca®. Your doctor or dietitian can also help you with developing a meal plan that is best for you.

Talk to your physician if you have any questions about Zavesca® or type 1 Gaucher disease.

Important Safety Information for Zavesca®:
In clinical studies, the most common adverse events due to Zavesca® included weight loss, diarrhea, and trembling in the hand (tremor). Other common adverse reactions were excess gas (flatulence), abdominal pain, headache, and influenza-like symptoms. The most common serious adverse reaction was tingling or numbness in the hands or feet with or without pain (peripheral neuropathy). Patients should undergo neurological examination at the start of treatment and every 6 months thereafter; Zavesca® should be reassessed in patients who develop symptoms of peripheral neuropathy. Zavesca® may cause fetal harm if administered to a pregnant woman. Men should maintain reliable contraceptive methods and not plan to father a child while taking Zavesca® and for 3 months after discontinuing treatment.

Please see full prescribing information. (the link behind full prescribing information is www.zavesca.com)

1. Zavesca® (miglustat) full prescribing information. Actelion Pharmaceuticals US, Inc. 2008.
http://www.zavesca.com/pdf/Zavesca_Prescribing_Information.pdf