iptacopan hydrochloride

Iptacopan hydrochloride (formerly LNP023, marketed under the brand name Fabhalta) is a first-in-class, orally active, highly selective small-molecule inhibitor of complement factor B (FB), a key serine protease in the alternative complement pathway. Developed by Novartis, iptacopan binds to the active site of factor B, preventing the cleavage of C3 and the subsequent assembly of the alternative pathway C3 convertase (C3bBb). This proximal inhibition effectively controls both C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis. Iptacopan is FDA-approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), primary immunoglobulin A nephropathy (IgAN), and complement 3 glomerulopathy (C3G). It is also undergoing clinical evaluation for other complement-mediated conditions, including atypical hemolytic uremic syndrome (aHUS), immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN), lupus nephritis, and generalized myasthenia gravis.

Brand names

Fabhalta飞赫达

Other names

ipracopanipracopan hydrochlorideiptacopaniptacopan hydrochloride monohydrate盐酸伊普可泮