pabinafusp alfa

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Ligands
pabinafusp alfa

GtoPdb Ligand ID: 11772

Synonyms: IZCARGO® | JR-141 | JR141

pabinafusp alfa is an approved drug (Japan (2021))

Comment: Pabinafusp (JR-141) is a fusion protein of the Fab domains of an anti-transferrin receptor monoclonal and the enzyme iduronate 2-sulfatase (IDS; P22304) [5], an enzyme that is deficient in patients with the lysosomal storage disease mucopolysaccharidosis type II (MPS II; Hunter syndrome) [1-2]. It is an intravenously administered enzyme replacement therapy. The anti-transferrin receptor component facilitates receptor-mediated movement of the fused enzyme across the blood-brain barrier [4].

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Summary of Clinical Use
Pabinafusp is approved in Japan, as an enzyme replacement therapy for patients with mucopolysaccharidosis type II (MPS II), and in particular for those for whom existing therapies have been ineffective. Exogenously delivered iduronate-2-sulfatase to the brain is intended to combat the neurocognitive impairment and slow progressive neurodegeneration in MPS II patients. Data from a phase 2/3 study suggests that this therapy may also improve peripheral symptoms [3].

Summary of Clinical Use

Pabinafusp is approved in Japan, as an enzyme replacement therapy for patients with mucopolysaccharidosis type II (MPS II), and in particular for those for whom existing therapies have been ineffective. Exogenously delivered iduronate-2-sulfatase to the brain is intended to combat the neurocognitive impairment and slow progressive neurodegeneration in MPS II patients. Data from a phase 2/3 study suggests that this therapy may also improve peripheral symptoms [3].

Clinical Trials
Clinical Trial ID	Title	Type	Source	Comment	References
NCT03568175	A Study of JR-141 in Patients With Mucopolysaccharidosis II	Phase 2/Phase 3 Interventional	JCR Pharmaceuticals Co., Ltd.		3
NCT04573023	A Phase Ⅲ Study of JR-141 in Patients With Mucopolysaccharidosis II	Phase 3 Interventional	JCR Pharmaceuticals Co., Ltd.