What is the mechanism of action of miglustat?

Mode of action Miglustat is a small iminosugar molecule that acts as a competitive inhibitor of the enzyme glucosylceramide synthase, which catalyzes the first committed step in glycosphingolipid (GSL) synthesis, the glycosylation of ceramide.

What does miglustat inhibit?

Miglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase.

How does substrate reduction therapy work?

Substrate reduction therapy is a new approach in which glycolipid accumulation is counteracted not by replacing the deficient enzyme but by reducing the substrate level to better balance residual activity of the deficient enzyme. Miglustat is an inhibitor of glucosylceramide synthase, a key enzyme in GSL synthesis.

Who makes Zavesca?

Zavesca (miglustat) is a substrate reduction therapy marketed by Actelion Pharmaceuticals for the treatment of mild to moderate type 1 Gaucher disease.

What is miglustat used for?

Miglustat is used to treat Gaucher disease type 1 (a condition in which a certain fatty substance is not broken down normally in the body and instead builds up in some organs and causes liver, spleen, bone, and blood problems). Miglustat is in a class of medications called enzyme inhibitors.

Who makes miglustat?

Miglustat, sold under the brand name Zavesca, is a medication used to treat type I Gaucher disease (GD1). It is also known as N-butyldeoxynojirimycin, and is a derivative of the anti-diabetic 1-deoxynojirimycin. It was developed by Oxford GlycoSciences and is marketed by Actelion.

How is vpriv made?

The active ingredient of VPRIV is velaglucerase alfa, which is produced by gene activation technology in a human fibroblast cell line. Velaglucerase alfa is a glycoprotein of 497 amino acids; with a molecular weight of approximately 63 kDa.

What is SRT medication?

Substrate reduction therapy (SRT) is an oral medication for Gaucher disease (pronounced go-SHAY). SRT is not available for women who are pregnant or breastfeeding, nor is it approved for use in children. Learn more about Gaucher disease treatment options.

Is miglustat FDA approved for NPC?

Miglustat has been approved in the EU, Canada, and Japan for treating progressive neurological complications in people with Niemann–Pick disease, type C (NPC). Miglustat has not received approval for this indication outside of these countries and use for this disease, including the US, is off-label.

Is miglustat generic?

Because Miglustat Dipharma is a generic medicine and is bioequivalent to the reference medicine, its benefits and risks are taken as being the same as the reference medicine’s.