Patient stories
View different patients' experiences living with type 1 Gaucher disease and taking Zavesca therapy.
Learn moreFAQ
What should you know about taking Zavesca? Find answers to your most common questions here.
Learn moreHow it works
Zavesca reduces harmful buildup
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 by reducing the amount of GSL the body produces.1* This way, the enzymes you do have are able to break down the fatty substances.
Understanding Your Zavesca Therapy is an Important First Step.
Click to download
Zavesca also helps increase the ability of natural enzymes in the body to work more effectively in its cells.2
Key benefits
In people with type 1 Gaucher disease, the buildup of fatty substances (GSLs) often causes swelling of and damage to the liver and spleen and many different types of bone problems. People may also have lowered levels of hemoglobin (a substance in red blood cells) and platelets (blood-clotting cells) that may cause anemia (low red blood cell count), nosebleeds, and bruising.
Zavesca reduces the harmful buildup of glycosphingolipids throughout the body to help treat the symptoms of type 1 Gaucher disease.
Clinical studies have shown that Zavesca provides 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 spleen1
- Increases amounts of hemoglobin and platelets1
*Based on laboratory studies
What is ZAVESCA?
ZAVESCA is a prescription medicine taken by mouth for adults with mild to moderate type 1 Gaucher disease. ZAVESCA is used only in people who cannot be treated with enzyme replacement therapy.
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, and headache. 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.
REF:
| 1. | Zavesca® (miglustat) full prescribing information. Actelion Pharmaceuticals US, Inc. 2010. |
|---|---|
| 2. | Alfonso P, Pampín S, Estrada J, et al. Miglustat (NB-DNJ) works as a chaperone for mutated acid ß-glucosidase in cells transfected with several Gaucher disease mutations. Blood Cell Mol. Dis. 2005;35:268-276. |
