Other Miscellaneous Therapeutic Agents; Enzyme Inhibitor. (ATC-Code: A16AX06).
Mechanism of action
Miglustat competitively and reversibly inhibits enzyme needed to produce glycosphingolipids and decreases rate of glycosphingolipid glucosylceramide formation.
Therapeutic use
Treatment of mild-to-moderate type 1 Gaucher’s disease when enzyme replacement therapy not a therapeutic option.
Pregnancy and lactiation implications
Decreased fetus weight, fetal loss, and difficult or delayed births observed in animal studies. Not recommended during lactation.
Unlabeled use
Contraindications
Hypersensitivity to miglustat or any component of the formulation. Pregnancy.
Warnings and precautions
Diarrhea common. Peripheral neuropathy reported. Exacerbations of existing tremor or tremor might occur. Use with caution in renal impairment. Not recommended in severe impairment. Miglustat increases clearance of imiglucerase.