Brands, Medical Use, Clinical Data
Brands / Synonyms
Agalsidase alfa; Alpha-D- galactoside galactohydrolase; Alpha-D-galactosidase A; Alpha-galactosidase A precursor; Fabrazyme; Fabrazyme (Genzyme Corp)
Recombinant human alpha-galactosidase A. The mature protein is composed of 2 subunits of 398 residues. Protein is glycosylated and produced by CHO cells
For treatment of Fabry's disease (alpha-galactosidase A deficiency)
Used in the treatment of Fabry disease, an X-linked genetic disorder of glycosphingolipid metabolism. The disease is characterized by a deficiency of the lysosomal enzyme alpha-galactosidase A, which leads to progressive accumulation of glycosphingolipids, predominantly GL-3, in many body tissues. Clinical manifestations of Fabry disease include renal failure, cardiomyopathy, and cerebrovascular accidents. Fabrazyme is intended to provide an exogenous source of alpha-galactosidase A and to limit the accumulation of these glycolipids in the tissues.
Mechanism of Action
Alpha-galactosidase A catalyzes the hydrolysis of globotriaosylceramide (GL-3) and other a-galactyl-terminated neutral glycosphingolipids, such as galabiosylceramide and blood group B substances to ceramide dihexoside and galactose.
Biotrnasformation / Drug Metabolism