idursulfase

Idursulfase is a purified recombinant form of the human lysosomal enzyme iduronate-2-sulfatase, produced using recombinant DNA technology in a human cell line. It is used as an enzyme replacement therapy (ERT) for Hunter syndrome (Mucopolysaccharidosis II, MPS II), a rare X-linked recessive disorder caused by deficiency of the endogenous iduronate-2-sulfatase enzyme. The absence or malfunction of this enzyme leads to accumulation of glycosaminoglycans (GAGs), specifically dermatan sulfate and heparan sulfate, in various tissues and organs. Idursulfase works by providing exogenous functional enzyme that is taken up into cellular lysosomes via mannose-6-phosphate receptor-mediated endocytosis, allowing catabolism of accumulated GAGs and reducing disease symptoms such as organomegaly and impaired mobility. It does not cure Hunter syndrome but can improve walking ability and reduce spleen size in affected patients[1][3][4][7].

Brand names

Elaprase

Other names

intrathecal idursulfase