Daily Medication Pearl: Miglustat (Zavesca)


Miglustat (Zavesca) is indicated for the treatment of mild to moderate type I Gaucher disease.

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.


  • 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


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