12.1 Mechanism of Action Hunter syndrome is an inherited X-linked recessive lysosomal storage disease caused by a deficiency of iduronate-2-sulfatase (IDS), a lysosomal enzyme, that degrades heparan sulfate (HS) and dermatan sulfate (DS), the two primary glycosominoglycans (GAGs) in the lysosome. Insufficiency or absence of IDS leads to accumulation of GAGs, including HS and DS, and subsequent lysosome dysfunction in multiple organs and tissues, including the central nervous system (CNS). Tividenofusp alfa-eknm provides an exogenous source of IDS. The fragment, crystallizable (Fc) component of tividenofusp alfa-eknm binds to the apical domain of the transferrin receptor (TfR) and delivers IDS to peripheral tissues and to the CNS through receptor-mediated transcytosis across the blood-brain barrier. Tividenofusp alfa-eknm is internalized via binding to the mannose-6-phosphate receptor on the cell surface and transported into lysosomes where it is thought to exert enzymatic activity and reduce accumulated GAGs. In addition, since TfR is ubiquitously expressed, it is expected that the interaction of tividenofusp alfa-eknm and TfR will contribute to its uptake into cells in the brain and peripheral tissues.

indication

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

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tividenofusp alfa

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AVLAYAH (TIVIDENOFUSP ALFA-EKNM) — FDA approval

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Avlayah

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tividenofusp alfa

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Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

phase

marketed

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