miglustat

Miglustat is a small molecule inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in glycosphingolipid synthesis. It is used primarily as an oral therapy for adults with mild to moderate type 1 Gaucher disease who cannot be treated with enzyme replacement therapy (ERT), and in combination with cipaglucosidase alfa-atga (Pombilti) for late-onset Pompe disease in adults not responding adequately to ERT. Miglustat reduces the accumulation of harmful glycosphingolipids by inhibiting their synthesis, thereby alleviating symptoms associated with substrate buildup such as organomegaly and bone complications. It has also been approved in some regions for Niemann-Pick disease type C[1][2][3][4][5].

Brand names

ZavescaYargesaOpfolda

Other names

N-butyl-deoxynojirimycinN-butylmoranolinen-butyl deoxynojirimycinbutyldeoxynojirimycinmiglustatum