agalsidase beta

Agalsidase beta is a recombinant human alpha-galactosidase A used as enzyme replacement therapy for the treatment of Fabry disease, a rare inherited lysosomal storage disorder caused by deficiency of the enzyme alpha-galactosidase A. This deficiency leads to accumulation of globotriaosylceramide and other glycosphingolipids in various tissues, resulting in progressive organ damage. Agalsidase beta works by replacing the missing or deficient enzyme, thereby hydrolyzing accumulated substrates and reducing or preventing disease manifestations such as renal failure, cardiomyopathy, and cerebrovascular events. The drug is administered intravenously every two weeks and is indicated for use in adults and children 2 years of age and older with confirmed Fabry disease[1][2][3][4][5][8].

Brand names

Fabrazyme

Other names

recombinant human alpha-galactosidase A