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

The treatment is also being investigated in patients with type 3B Gaucher disease in combination with enzyme replacement therapy.

The U.S. Food and Drug Administration (FDA) approved the use of Zavesca in 2003, followed by the approval of generic miglustat in 2018. The European Medicines Agency (EMA) has granted marketing authorization for Zavesca in the European Union.

How Zavesca works

In type 1 Gaucher disease, the body’s ability to break down a fatty chemical called glucocerebroside is affected due to deficiency of an enzyme called beta-glucocerebrosidase. This causes glucocerebroside to build up in the spleen, liver, and bone marrow, leading to many complications.

Zavesca is a treatment option for patients for whom enzyme replacement therapy, where additional beta-glucocerebrosidase is directly administered into the body, cannot be used due to various reasons such as allergy and hypersensitivity. It works by preventing the further synthesis of glucocerebroside by inhibiting an enzyme called glucosylceramide synthase, thereby reducing the enlargement of organs and other symptoms of type 1 Gaucher disease.

Zavesca in clinical trials

A one-year open-label trial to study the effect of Zaveska in type 1 Gaucher disease was completed in 2000. A 19% reduction in liver and spleen volumes was observed, with diarrhea being the most frequent side effect. The FDA’s approval of Zavesca was based on the results of this trial.

An open-label, multicenter Phase 3 trial (NCT00319046) to study the long-term safety and efficacy of Zavesca in adults with type 1 Gaucher disease was completed in 2010. The results showed that treatment with Zavesca was able to maintain overall liver size. Although some patients experienced a deterioration in symptoms, findings showed that Zavesca could maintain clinical stability in others.

A clinical trial (NCT02520934) is currently evaluating the effects of the combination of Zavesca treatment and enzyme replacement therapy in patients with type 3B Gaucher disease. The study is estimated to be completed in December 2019.

Other information

It is advised that Zavesca be administered by physicians with knowledge about the management of patients with Gaucher disease.

If adverse reactions such as tremor or diarrhea are seen, the dosage may be reduced according to the physician’s recommendation. A mild reduction in platelet counts might also be observed in some patients.

Zavesca can also be used for the treatment of Neimann-Pick disease type C.


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