Miglustat

Miglustat is a drug used to treat Gaucher disease.

It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids.

It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase.

It is marketed under the trade name Zavesca.

It has also been investigated for use in treating Niemann-Pick disease.