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

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

Acknowledgments

The author would like to thank the patients with Gaucher's disease and their physicians and healthcare personnel who submit data to the ICGG Gaucher Registry and the Gaucher Registry support team at the Genzyme Corporation. Further thanks are due to Andrea Gwosdow (PhD) for assistance in preparing the manuscript. Finally, the author thanks Carolyn Sawyer (PhD) and Susan Graham (BSc, MSc and MBChB) for critical review of the manuscript, Tim Edmunds (PhD) for scientific discussion and helpful suggestions and Robert Brown and Trent Richardson for graphic design of the figures, all of whom are at the Genzyme Corporation.

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