ABSTRACT
This chapter outlines the development of Ceredase and Cerezyme from the initial discovery of the enzymatic defect in Gaucher Disease to the approval of Cerezyme. Ceredase was a natural product of human origin, a higher level of host protein impurities was allowed than would be typical of a recombinant product. In developing the recombinant product, it was also desirable to eliminate the human serum albumin that was used as a stabilizer in Ceredase to reduce further the risk of viral contamination. The clinical trial that led to the approval of Ceredase was conducted on 12 patients, 4 adults and 8 children who were classified with Type I Gaucher Disease. Following the identification of ß-glucocerebrosidase as the defective enzyme in Gaucher Disease and the proposal that the disease could be treated by replacing the defective enzyme, it took several years until sufficient material of optimum purity was available for clinical studies.
