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Actelion/Celltech Zavesca

This article was originally published in Pharmaceutical Approvals Monthly

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Executive Summary

Actelion's type I Gaucher disease therapy Zavesca (miglustat) is approved July 31 as second-line treatment to Genzyme's Cerezyme (imiglucerase) in adults for whom "enzyme replacement therapy is not an option." The firm estimates the eligible population at 200-300 patients. The product will be available to a limited number of physicians due to neuropathy concerns and pregnancy category X status. Actelion and Celltech are studying miglustat in type III Gaucher disease, Niemann-Pick type C and late onset Tay-Sachs..

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This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

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