Agalsidase beta

Identification

Name
Agalsidase beta
Accession Number
DB00103 / RZD65TSM9U
Groups
Fabrazyme
Description

Recombinant human alpha-galactosidase A. The mature protein is composed of 2 subunits of 398 residues. Protein is glycosylated and produced by using CHO cells

Pharmacology

Pharmacodynamics

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. Studies show unconclusive evidence for the clinical efficacy of either agalasidase alfa or agalasidase beta in preventing morbidity.

Indication

For treatment of Fabry's disease (alpha-galactosidase A deficiency)

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.