alglucosidase alfa

Alglucosidase alfa is a recombinant form of the human enzyme acid alpha-glucosidase (GAA), produced using Chinese hamster ovary cell lines. It is used as an enzyme replacement therapy for Pompe disease (glycogen storage disease type II), a rare lysosomal storage disorder caused by GAA deficiency. The drug works by catalyzing the hydrolysis of α-1,4- and α-1,6-glycosidic linkages in lysosomal glycogen, thereby reducing glycogen accumulation in tissues. Alglucosidase alfa is administered intravenously and is indicated for both infantile-onset and late-onset Pompe disease[1][2][5][8].

Brand names

LumizymeMyozyme

Other names

acid alpha-glucosidaserecombinant human acid alpha-glucosidase