Force Majeure: Therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease

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• 作者：Carla E.M. Hollak ; Stephan vom Dahl ; Johannes M.F.G. Aerts ; Nadia Belmatoug ; Bruno Bembi ; Yossi Cohen ; Tanya Collin-Histed ; Patrick Deegan ; Laura van Dussen ; Pilar Giraldo ; Eugen Mengel ; Helen Michela
• 关键词：Gaucher disease ; Cerezyme ; Imiglucerase ; Vesivirus ; Europe
• 刊名：Blood Cells, Molecules, and Diseases
• 出版年：2010
• 出版时间：15 January 2010
• 年：2010
• 卷：44
• 期：1
• 页码：41-47
• 全文大小：175 K

文摘

Gaucher disease is the first lysosomal disorder for which clinically effective enzyme replacement therapy has been introduced. Lifelong treatment with imiglucerase, the recombinant glucocerebrosidase manufactured by the Genzyme Corporation (MA, USA), is administered intravenously — usually at biweekly intervals. An acute shortage of imiglucerase (to 20 % of prior global supply) has occurred as a result of viral contamination of the production facility; production was halted, and a full supply of imiglucerase is not anticipated until January 2010. An urgent meeting of physicians, researchers, and patients was convened through the agency of the European Working Group for Gaucher Disease; this was instigated by patients internationally represented by the European Gaucher Alliance. Here we present a position statement based on the findings of the group, with key recommendations about identification and monitoring of at-risk patients threatened by the abrupt withdrawal of treatment, the equitable distribution of residual imiglucerase — and access to alternative treatments including those that have completed phase III clinical trials but have not yet been licensed.