Miglustat reduces the formation of a certain protein in the body in people with type 1 Gaucher disease. 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.
Zavesca has an active substance called miglustat that prevents an enzyme known as glucosylceramide synthase from working. This enzyme is needed in the preliminary phase of the production of glycosphingolipids in the body cells. By halting the enzyme from working, miglustat reduces the production of glycosphingolipids in the cells. This results in slowing down or preventing the development of signs of type-1 Gaucher ailment and to reduce the symptoms of Niemann-Pick type-C sickness.