First oral treatment option for type 1 Gaucher disease
The FDA has approved Actelion's Zavesca (miglustat) capsules as the first oral treatment option for type 1 Gaucher disease.
The FDA has approved Actelion's Zavesca (miglustat) capsules as the first oral treatment option for type 1 Gaucher disease.
Type 1 Gaucher disease is a rare genetic lipid storage disorder affecting an estimated 10,000 individuals worldwide. It is a progressive condition that is caused by a deficiency of glucocerbrosidase, an im-portant enzyme in the metabolism of key lipids in the body. The deficiency of this enzyme results in accumulation of excess amounts of glycosphingolipids (GSLs) in specific cells primarily in the liver, spleen and bone marrow. Such accumulation leads to liver and spleen enlargement/dysfunction, anemia, bone disease and pain.
Current therapy includes enzyme replacement therapy (ERT), which is delivered via an intravenous infusion twice monthly.
Zavesca is the first in a new class of drugs known as substrate reduction therapy (SRT), which reduces the amount of glycosphingolipid (GSL) production to a level which can effectively cleared by the naturally occurring glucocerbrosidase in the cells. Zavesca will be available to patients in the United States later this year.
The FDA has approved Zavesca for the treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access).
Zavesca is approved in the European Union and is commercially available in the United Kingdom and Germany.
Background on Actelion and Zavesca
Actelion is the license holder for Zavesca worldwide, with the exception of Israel where the drug is also approved. Actelion is responsible for all regulatory and marketing activities and will book all sales of Zavesca. The drug was originally developed by Oxford GlycoSciences (OGS), now part of the Celltech Group. Through a joint steering committee, Actelion and Celltech are currently conducting clinical studies with Zavesca for the treatment of other lipid storage disorders, such as type 3 Gaucher disease, Niemann-Pick type C, and Late Onset Tay-Sachs.