Miglustat

Definition

A synthetic, N-alkylated imino analogue of D-glucose. Miglustat competitively and reversibly binds to and inhibits the activity of UDP-glucose ceramide glucosyltransferase, which catalyzes the initial step in the synthesis of glycosphingolipids (GSL). This leads to a decrease in the production of glycosphingolipid, which has important roles in various cellular processes. Miglustat can be used in substrate reduction therapy in diseases in which the enzyme glucocerebrosidase, that is responsible for the breakdown of GSL, is deficient.

Also known as 1,5-(butylimino)-1,5-dideoxy-D-glucitol, 3,4,5-Piperidinetriol, 1-butyl-2-(hydroxymethyl)-,(2R-(2alpha,3beta,4alpha,5beta))-, MIGLUSTAT, N-Butyl Deoxynojirimycin, N-Butylmoranoline, SC 48334, SC-48334, Zavesca — per NCIT

Also identified as

• ATC A16AX06 per RXNORM
• NCIT NCIT:C1222 per NCIT
• SCTID 408044003 per RXNORM
• SCTID 412296005 per RXNORM
• UNII ADN3S497AZ per RXNORM

Indications