Cerezyme^® (imiglucerase for injection)

Bibliographical Information:

Drug Name	Generic Name	Studied Indications or Disease	Approved U.S. Drug Label
Cerezyme®	imiglucerase for injection	Type 1 Gaucher disease	Prescribing Info

Protocol Number RC 91-0110

[Summary of original Ceredase^®* clinical study: Barton, NW et al. 1991. “Replacement Therapy for Inherited Enzyme Deficiency-Macrophage Targeted Glucocerebrosidase for Gaucher disease.” New England Journal of Medicine 324:1464-1470. ]

Protocol RC 92-0301

Zimran A, Elstein D, Levy-Lahad E, Zevin S, Hadas-Halpern I, Bar-Ziv Y, et al. 1995. “Replacement therapy with imiglucerase for type 1 Gaucher's disease.” Lancet 345(8963):1479-80.

Protocol RC 92-0501

Grabowski GA, Barton NW, Pastores G, et al. 1995. “Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources.” Ann Intern Med 122(1):33-9.

Pastores GM. 1998. “Recombinant Enzyme Therapy for Gaucher disease: long-term clinical experience and therapeutic outcomes with cross-over from alglucerase.” Gaucher Clinical

Perspectives: Molecular Medicine and Therapeutics 6(1):5-8.

Hermann G, Pastores GM, Abdelwahab IF, Lorberboym AM. 1997. Gaucher disease: assessment of skeletal involvement and therapeutic responses to enzyme replacement. Skeletal Radiol 26(12):687-96.

*Ceredase^® (alglucerase injection) is human placental-derived glucocerebrosidase, which is the original enzyme replacement therapy for Type I Gaucher disease and has largely been replaced by recombinant human glucocerebrosidase, Cerezyme^® (imiglucerase for injection).