Daily Medication Pearl: Miglustat (Zavesca)

Medication Pearl of the Day: Miglustat (Zavesca)

Indication: Miglustat is an inhibitor of the enzyme glucosylceramide synthase, which is a glucosyl transferase enzyme responsible for the first step in the synthesis of most glycosphingolipids. The drug is indicated for the treatment of mild to moderate type I Gaucher disease.

Insight:

• Dosing:Treatment with miglustat as monotherapy at a starting dose of 100 mg, 3 times daily (dosage range 100 mg once daily to 200 mg 3 times daily) in adult patients with type 1 Gaucher disease.
• Dosage forms: 100 mg capsule.
• Adverse events: Diarrhea, flatulence, nausea, vomiting, anorexia and others.
• Mechanism of action: The goal of treatment is to reduce the rate of glycosphingolipid biosynthesis so that the amount of glycosphingolipid substrate is reduced to a level that allows the residual activity of the deficient glucocerebrosidase enzyme to be more effective.
• Manufacturer: Janssen

Sources:

• label (fda.gov)
• ZAVESCA® (miglustat): For the treatment of type 1 Gaucher Disease
• Zavesca-USPI_20210115_NDA_Clean[1] (janssenlabels.com)