Agalsidase Beta

Fabry disease arises from deficient alpha-galactosidase A activity, and agalsidase beta is the recombinant enzyme used to supply what the patient cannot make. Administered by infusion, it restores hydrolysis of globotriaosylceramide, the glycosphingolipid that otherwise accumulates in the vascular endothelium, kidney, heart and other tissues. Reducing that substrate burden slows the progression of renal damage and the associated cardiac and cerebrovascular complications of the disorder. As a recombinant protein product rather than a small molecule, its pharmacology is that of enzyme replacement: it substitutes for deficient endogenous production and re-establishes a normal metabolic step instead of blocking a receptor or transporter. The agent is approved and has reached the final stage of clinical development.