Miglustat (4 brand names, 1 dosage form, 1 route)
Available dosage forms: CAPSULE.
Routes of administration: ORAL.
Brand Names
- MIGLUSTAT
- OPFOLDA
- YARGESA
- ZAVESCA
Contraindications
None.
Adverse Reactions
The following serious adverse reactions are described below and elsewhere in the labeling: Peripheral Neuropathy [see Warnings and Precautions (5.1)] Tremor [see Warnings and Precautions (5.2)] Diarrhea and Weight Loss [see Warnings and Precautions (5.3)] Reductions in Platelet Count [see Warnings and Precautions (5.4)]
Drug Interactions
While co-administration of miglustat appeared to increase the clearance of imiglucerase by 70%, these results are not conclusive because of the small number of patients studied and because patients took variable doses of imiglucerase [see Clinical Pharmacology (12.3)].
Mechanism of Action
Type 1 Gaucher disease is caused by a functional deficiency of glucocerebrosidase, the enzyme that mediates the degradation of the glycosphingolipid glucosylceramide. Miglustat functions as a competitive and reversible inhibitor of the enzyme glucosylceramide synthase, the initial enzyme in a series of reactions which results in the synthesis of most glycosphingolipids. Miglustat helps reduce the rate of glycosphingolipid biosynthesis so that the amount of glycosphingolipid substrate is reduced to a level which allows the residual activity of the deficient glucocerebrosidase enzyme to be more effective (substrate reduction therapy). In vitro and in vivo studies have shown that miglustat can reduce the synthesis of glucosylceramide-based glycosphingolipids.