vimircopan

Vimircopan (BCX9930) is an orally bioavailable, potent, and selective small-molecule inhibitor of complement factor D (CFD), a serine protease that serves as the rate-limiting enzyme in the alternative pathway of the complement system. Developed by BioCryst Pharmaceuticals, vimircopan was designed to treat various complement-mediated diseases by preventing the formation of C3 convertase, thereby inhibiting the amplification of the complement cascade. It was primarily evaluated in Phase 2 clinical trials for paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and IgA nephropathy. In 2023, BioCryst announced the discontinuation of the BCX9930 program to focus on other assets, despite observing clinical proof-of-concept. Recent academic research has also explored its potential in treating pulmonary hypertension by targeting fibroblast-specific complement activation.

Brand names

pelecopan

Other names

pelecopan