Miglustat (Zavesca) is an innovative glucosylceramide synthase inhibitor, which was first approved in the United States in 2003.
What Kind of Medicine is Miglustat (Zavesca)?
Main Therapeutic Areas
Miglustat is a glucosylceramide synthase inhibitor, used as a monotherapy for the treatment of adult patients with mild to moderate Type 1 Gaucher disease.
It is indicated for patients for whom enzyme replacement therapy is not a treatment option (e.g., due to allergy, hypersensitivity, or poor venous access).
Pharmacological Actions
Type 1 Gaucher disease is caused by a functional deficiency of glucocerebrosidase, an enzyme that mediates the degradation of the glycosphingolipid glucosylceramide.
Miglustat acts as a competitive and reversible inhibitor of glucosylceramide synthase, which is the initial enzyme in the series of reactions for the synthesis of most glycosphingolipids.
Miglustat helps reduce the rate of glycosphingolipid biosynthesis, lowering the amount of glycosphingolipid substrates to a level that allows the residual activity of the defective glucocerebrosidase to function more effectively (substrate reduction therapy).
Dosage Form, Strength and Characteristics of Miglustat (Zavesca)
Details of Dosage Form and Strength
Miglustat is available as 100 mg capsules, each containing 100 mg of miglustat.
The capsules are white, opaque, hard gelatin capsules, with "OGT918" printed in black on the cap and "100" printed in black on the body.
Chemical Composition Analysis
The chemical name of miglustat is 1,5-(butylimino)-1,5-dideoxy-D-glucitol, with a chemical formula of C₁₀H₂₁NO₄ and a molecular weight of 219.28.
Miglustat is a white to off-white crystalline solid with a bitter taste. It is highly soluble in water (>1000 mg/mL as the free base).
Excipient Composition Analysis
Capsule contents: Sodium starch glycolate, povidone (K30), magnesium stearate.
Capsule shell materials: Gelatin, titanium dioxide, edible ink (containing black iron oxide and shellac).
Storage Method of Miglustat (Zavesca)
Standard Storage Conditions
Miglustat must be stored at 20°C to 25°C (68°F to 77°F), with allowable short-term fluctuations between 15°C and 30°C (59°F and 86°F).
Packaging and Supply Features
Miglustat is packaged in blister cards.
NDC 66215-201-90: Each carton contains 90 capsules.
NDC 66215-201-15: Each blister card contains 15 capsules.
Key Storage Points
The medicine must be kept in a controlled room temperature environment.
Ensure the medicine is always stored out of the reach of children.
Keep the medicine in its original packaging.
