vestronidase alfa

Vestronidase alfa is a recombinant human lysosomal beta-glucuronidase used as an enzyme replacement therapy for the treatment of mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. MPS VII is a rare, inherited metabolic disorder caused by deficiency of the enzyme beta-glucuronidase, leading to accumulation of glycosaminoglycans (GAGs) in tissues and organs. Vestronidase alfa acts by providing an exogenous source of beta-glucuronidase, which is taken up into cellular lysosomes via mannose-6-phosphate receptor-mediated endocytosis. This enables catabolism of accumulated GAGs in affected tissues, reducing their toxic buildup and improving clinical symptoms such as mobility and organ function[1][2][6]. The drug was developed using recombinant DNA technology in Chinese hamster ovary cells and has the same amino acid sequence as human beta-glucuronidase[2][6]. It does not cure MPS VII but can improve certain symptoms and quality of life for patients[5].

Brand names

Mepsevii

Other names

vestronidase alfa-vjbk