vemircopan

Vemircopan is an orally bioavailable small molecule inhibitor of complement factor D, a serine protease that cleaves complement factor B in the alternative pathway of the complement cascade. By inhibiting complement factor D, vemircopan blocks activation of the alternative pathway, thereby preventing downstream effects such as hemolysis and tissue damage associated with diseases like paroxysmal nocturnal hemoglobinuria (PNH). Vemircopan was originally developed by Achillion Pharmaceuticals and is currently being developed by Alexion AstraZeneca Rare Disease. It has been investigated primarily for PNH but also for other indications including IgA nephropathy, lupus nephritis, generalized myasthenia gravis, and C3 glomerulopathy (dense deposit disease). The drug has orphan designation for PNH but several development programs have been discontinued[1][2][5][6][7].

Brand names

vemircopan

Other names

vemircopan [INN]2086178-00-7