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.
Miglustat is a generic medication commonly marketed under the brand name Zavesca. Miglustat belongs to a class of drugs known as Miscellaneous metabolic agents medication. Miglustat medication is used to treat adult patients who have mild to moderate type 1 Gaucher disease. Safety of Miglustat medication for pediatric patients those below the age of 18 has not yet been established.