pabinafusp alfa

Pabinafusp alfa is a recombinant fusion protein composed of human iduronate-2-sulfatase (IDS) genetically fused with a humanized anti-human transferrin receptor antibody. It is designed as an enzyme replacement therapy for mucopolysaccharidosis II (MPS II, also known as Hunter syndrome), a rare X-linked lysosomal storage disorder caused by deficiency of IDS. Unlike conventional ERTs, pabinafusp alfa can cross the blood-brain barrier via transferrin receptor-mediated transcytosis, enabling it to address both central nervous system and peripheral symptoms of MPS II. Clinical studies have shown that intravenous administration reduces heparan sulfate concentrations in cerebrospinal fluid and improves or stabilizes neurocognitive development in affected patients. Pabinafusp alfa was approved in Japan in March 2021 and represents the first BBB-penetrating enzyme therapy for neuronopathic MPS II[1][3][5][6][8].

Brand names

IZCARGO

Other names

pabinafusp alfaJR-141JR141JR 141IZCARGO