laronidase

Laronidase is a recombinant form of the human enzyme alpha-L-iduronidase, produced using Chinese hamster ovary cell lines. It is a glycoprotein with a molecular weight of approximately 83 kDa and contains 6 N-linked oligosaccharide modification sites. Laronidase is used as an enzyme replacement therapy for patients with mucopolysaccharidosis I (MPS I), specifically the Hurler, Hurler-Scheie, and moderate to severe Scheie forms. MPS I results from a deficiency in endogenous alpha-L-iduronidase, leading to accumulation of glycosaminoglycans (GAGs) such as dermatan sulfate and heparan sulfate in tissues and organs. Laronidase catalyzes the hydrolysis of terminal alpha-L-iduronic acid residues from these GAGs, reducing their pathological accumulation. The drug has been shown to improve pulmonary function and walking capacity but does not address central nervous system manifestations due to limited blood-brain barrier penetration[1][3][4][5].

Brand names

Aldurazyme

Other names

recombinant human alpha-L-iduronidase