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Information about Miglustat

Miglustat is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease.

Liver safety of Miglustat

Clinical experience with miglustat is limited, but it has not been linked to serum enzyme elevations during therapy or to instances of clinically apparent acute liver injury.

Mechanism of action of Miglustat

Miglustat (me' gloo stat) is a small molecule inhibitor of glucosylceramide synthase, the first and rate controlling step in the pathway of glycolipid synthesis. By inhibiting the pathway, lower levels of substrate are available, less is available for lysosomal degradation and less glycosylceramide accumulates. Miglustat was shown to decrease the intracellular accumulation of glycosylceramide, the major glycolipid that accumulates in Gaucher disease, in animal models of the genetic disease. In several randomized controlled trials, miglustat was shown to decrease spleen and liver volume and increase hemoglobin and platelet counts in patients with type 1 Gaucher disease. Miglustat was also able to maintain clinical benefit in patients who had been maintained on long term enzyme replacement therapy with glucocerebrosidase infusions (the lysosomal enzyme that is deficient in type 1 Gaucher disease).

FDA approval information for Miglustat

Miglustat was approved in the United States in 2003 as oral therapy of type 1 Gaucher Disease in patients who are not eligible for enzyme replacement therapy. Miglustat has also been evaluated in other lysosomal enzyme deficiencies and is approved in Europe, but not the United States as therapy of Niemann–Pick disease, type C. Miglustat is available in tablets of 100 mg under the brand name Zavesca.

Dosage and administration for Miglustat

The usual dose regimen is 100 mg three times daily, but dose adjustments are needed for patients with significant side effects or renal impairment.

Side effects of Miglustat

Side effects are common and can be problematic including diarrhea, weight loss, gastrointestinal upset, nausea and vomiting, anorexia, constipation, headache, tremor, dizziness, weakness, visual problems, dry mouth, paresthesia, peripheral neuropathy, ataxia and memory loss.

genetic disorder agents

cystic fibrosis agents

enzyme replacement therapy

glucosylceramide synthase inhibitors (substrate restriction therapy)

lysosomal acid lipase deficiency agents


homocystinuria agents

Huntington disease agents

Monoclonal Antibodies

Tyrosinemia Agents

Urea Cycle Disorder Agents

Hematologic Agents

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