Fabrazyme^® (agalsidase beta)

Bibliographical Information:

Drug Name	Generic Name	Studied Indications or Disease	Approved U.S. Drug Label
Fabrazyme®	agalsidase beta	Fabry disease	Prescribing Info

Phase 1/2 (FB9702-01)

Desnick RJ, Brady R, Barranger J, Collins AJ, Germain DP, Goldman M, et al. Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy. Ann Intern Med 2003;138(4):338-46.

Eng, CM, Banikazemi, M; Gordon, RE; et al. A Phase 1/2 Clinical Trial of Enzyme Replacement in Fabry Disease: Pharmacokinetic, Substrate Clearance and Safety Studies. Am J Hum Genet. 2001:68:711-722.

Mignani R, Cagnoli L. Enzyme replacement therapy in Fabry's disease: recent advances and clinical applications. J Nephrol 2004;17(3):354-63.

Pastores GM, Thadhani R. Advances in the management of Anderson-Fabry disease: enzyme replacement therapy. Expert Opin Biol Ther 2002;2(3):325-33.

Phase 1/2 Extension (AGAL-006-99)

Desnick RJ, Brady R, Barranger J, Collins AJ, Germain DP, Goldman M, et al. Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy. Ann Intern Med 2003;138(4):338-46.

Phase 3 (AGAL-1-002-98)

Barbey F, Hayoz D, Widmer U, Burnier M. Efficacy of enzyme replacement therapy in Fabry disease. Curr Med Chem Cardiovasc Hematol Agents 2004;2(4):277-86.

Brenner B, Grunfeld J. Renoprotection by enzyme replacement therapy. Curr Opin Nephrol Hypertens 2004;13:231-41.

Breunig F, Wanner C. Enzyme replacement therapy for Fabry disease: proving the clinical benefit. Nephrol Dial Transplant 2003;18(1):7-9.

Desnick RJ, Brady R, Barranger J, Collins AJ, Germain DP, Goldman M, et al. Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy. Ann Intern Med 2003;138(4):338-46.

Eng CM; Guffon N; Wilcox WR; Germain DP, et al. Safety and Efficacy of recombinant human a-galactosidase A replacement therapy in Fabry’s disease. New England Journal of Medicine. 2001. 345.1: 9-16.

Hopkin RJ, Bissler J, Grabowski AG. Comparative evaluation of alpha-galactosidase A infusions for treatment of Fabry disease. Genet Med 2003;5(3):144-53.

Mignani R, Cagnoli L. Enzyme replacement therapy in Fabry's disease: recent advances and clinical applications. J Nephrol 2004;17(3):354-63.

Pastores GM, Thadhani R. Advances in the management of Anderson-Fabry disease: enzyme replacement therapy. Expert Opin Biol Ther 2002;2(3):325-33.

Siamopoulos KC. Fabry disease: kidney involvement and enzyme replacement therapy. Kidney International 2004;65(2):744-53.

Thurberg BL; Rennke H; Colvin RB, et al. Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy. Kidney International. 2002: 62:1933-1946.

Severe Renal (AGAL-022-02)

No associated publications