tividenofusp alfa

Tividenofusp alfa is an investigational recombinant fusion protein composed of the enzyme iduronate 2-sulfatase (IDS) fused to Denali Therapeutics' proprietary Enzyme Transport Vehicle (ETV). This design enables active transport of IDS across the blood-brain barrier and broad delivery throughout the body, aiming to address both central nervous system and somatic symptoms of Hunter syndrome (mucopolysaccharidosis type II, MPS II). The drug is being developed as a brain-penetrant enzyme replacement therapy for MPS II, a rare genetic lysosomal storage disorder caused by deficiency of IDS. Tividenofusp alfa has demonstrated normalization or near-normalization of key disease biomarkers in clinical trials and has shown improvements in cognitive, behavioral, auditory, and physical outcomes. It is currently under investigation in phase 2/3 clinical trials and has received FDA Breakthrough Therapy Designation as well as Fast Track status[1][3][4][5][6][7][8].

Brand names

AVLAYAH

Other names

iduronate-2-sulfatase fused to a Fc polypeptide that binds to the human transferrin receptoriduronate2-sulfatase fused to a Fc polypeptide that binds to the human transferrin receptoriduronate 2-sulfatase fused to a Fc polypeptide that binds to the human transferrin receptor