agalsidase alfa

Agalsidase alfa is a recombinant human alpha-galactosidase A enzyme used as enzyme replacement therapy for Fabry disease, a rare X-linked lysosomal storage disorder caused by deficiency of endogenous alpha-galactosidase A. The drug hydrolyzes globotriaosylceramide and other glycosphingolipids that accumulate in patients with Fabry disease, thereby preventing or reducing the severity of clinical manifestations such as renal failure, cardiomyopathy, and cerebrovascular events. Agalsidase alfa is taken up by cells via the mannose 6-phosphate receptor pathway. It was developed as an alternative to agalsidase beta and has been approved in Europe since 2001.

Brand names

Replagal

Other names

α-galactosidase Aalpha-galactosidase A