Imiglucerase and its use for the treatment of Gaucher's disease

Author: Weinreb, Neal J

Source: Expert Opinion on Pharmacotherapy, Volume 9, Number 11, August 2008 , pp. 1987-2000(14)

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Abstract:

Background: Gaucher's disease is caused by deficient lysosomal glucocerebrosidase activity. Intravenous enzyme replacement therapy with imiglucerase (Cerezyme^®, Genzyme Corporation, Cambridge, MA), a recombinant human glucocerebrosidase, ameliorates systemic manifestations such as hepatosplenomegaly, anemia, thrombocytopenia and skeletal abnormalities in patients with type 1 (non-neuronopathic) and type 3 (chronic neuronopathic) Gaucher's disease. Objective/methods: The aim of this study was to identify and comment on the current issues related to imiglucerase for Gaucher's disease based on a review of published English language literature and personal clinical experience. Results: The following topics were covered with respect to imiglucerase: development, pharmacokinetics, preparation and administration, efficacy, pediatrics, pregnancy, type 3 Gaucher's disease, dosing, treatment interruptions, safety and alternative pharmacological therapies. Conclusion: Imiglucerase is safe and well tolerated. In addition, it corrects the hepatic, splenic, hematologic and bone abnormalities observed with types 1 and 3 Gaucher's disease effectively and enhances health-related quality of life.

Keywords: enzyme replacement therapy; Gaucher's disease; imiglucerase; lysosomal storage disorders

Document Type: Research article

DOI: http://dx.doi.org/10.1517/14656566.9.11.1987

Publication date: 2008-08-01

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