Iptacopan

Generic Name: Iptacopan

Brand Names: Fabhalta

Drug Type: Small Molecule

Chemical Formula: C_₂₅H_₃₀N_₂O_₄

CAS Number: 1644670-37-0

Unique Ingredient Identifier: 8E05T07Z6W

Overview

Iptacopan is under investigation in clinical trial NCT04578834 (Study of Efficacy and Safety of LNP023 in Primary Iga Nephropathy Patients).

Background

Iptacopan is under investigation in clinical trial NCT04578834 (Study of Efficacy and Safety of LNP023 in Primary Iga Nephropathy Patients).

Indication

用于治疗既往未接受过补体抑制剂（如依库珠单抗等）治疗的阵发性睡眠性血红蛋白尿症(PNH，一种罕见病)成人患者。 PNH是一种由补体介导的罕见血液疾病。PNH患者的造血干细胞PIG-A基因发生突变，导致其产生易于被补体系统过早破坏的红细胞，从而引发血管内溶血（红细胞在血管内被破坏）和血管外溶血（红细胞在脾脏和肝脏中被破坏），临床主要表现为贫血、阵发性血红蛋白尿、骨髓造血功能衰竭和血栓形成等。

Associated Conditions

No associated conditions information available.

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CDSCO Approves Novartis' Protocol Amendment for Iptacopan Study in Rare Kidney Disorders

18 days ago

India's CDSCO expert committee has approved Novartis Healthcare's protocol amendment for a Phase IIIb extension study of iptacopan in patients with C3 glomerulopathy and idiopathic immune-complex-membranoproliferative glomerulonephritis.

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Drug Development Updates