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Clinical Development - Zavesca® |
THE ACTELION ANNUAL REPORT 2003 |
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| Clinical Development - Zavesca® |
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| Zavesca® trials ongoing |
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Zavesca® is registered for the treatment of type 1 Gaucher's disease, a genetically inherited disorder of lipid metabolism. Failure to metabolize glycosphingolipids leads to excess accumulation in cells, particularly in the liver, spleen and bone marrow – resulting in enlarged organs and marrow and bone abnormalities. Zavesca® provides a novel approach to the treatment of Gaucher’s disease by inhibiting the formation of glucosylceramide, an important element in the formation of these lipids. An added advantage of Zavesca® is that it is an oral treatment and therefore may be more suitable for those patients who are unable to receive enzyme replacement therapy.
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There is strong preclinical evidence to support the use of Zavesca® in other related lipid storage diseases for which currently no therapy exists. Consequently, Phase III trials in Niemann-Pick Type C, Late Onset Tay-Sachs and Type 3 Gaucher’s are being conducted. Results of these studies will be available in late 2004 and 2005.
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