Пэгунигалсидаза альфа – новый пегилированный рекомбинантный препарат альфа-галактозидазы для ферментозаместительной терапии болезни Фабри

Abstract

Fabry disease (FD) is a rare hereditary lysosomal storage disease. Enzyme replacement therapy improves symptoms and prevents progression of target organs involvement in patients with FD. Pegunigalsidase alfa is a novel, pegylated recombinant alpha-galactosidase enzyme characterized by prolonged half-life and reduced immunogenicity. Repeated kidney biopsy in phase 1/2 clinical trial demonstrated GL3 clearance from renal capillary endothelial cells. Three phase 3 clinical trials (BRIDGE, BRIGHT, and BALANCE) have shown favorable efficacy and safety profile in more than 130 patients with FD. In the pivotal phase 3 trial (BALANCE), pegunigalsidase alfa was non-inferior to agalsidase beta based on rate of eGFR decline over 2 years (the difference between median eGFR slopes was -0.36 mL/ min/1.73 m2/year) and was associated with a lower rate of infusion-related reactions in adult patients with Fabry disease and progressive nephropathy