Agalsidase_beta: profile and news

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Other information

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

Pharmacology
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 globotriaosy-lceramide (GL-3) and other a-galactyl-terminated neutral glycosphingolipids, such as galabiosylceramide and blood group B substances to ceramide dihexoside and galactose.

Drug Category
Enzyme replacement; enzymes (44:00.00)

Brand Names/Synonyms
Alpha-galactosidase A precursor;Melibiase;Alpha-D- galactoside galactohydrolase;Alpha-D-galactosidase A;Agalsidase alfa; Fabrazyme

Dosage Forms
POWDER FOR SOLUTION

Absorption
Not Available

Interactions
Not Available

Chemical IUPAC Name
Human alpha-galactosidase A.

Chemical Formula
C2029H3080N544O587S27

Half Life
45-102 min

Drug Type
Approved Drug

# Accession No
BIOD00075

CAS Registry Number
104138-64-9