Danicopan (ACH-4471, ALXN2040) is a potent, selective, reversible small-molecule inhibitor complement factor D with Kd of 0.54 nM.
Danicopan (ACH-4471, ALXN2040) inhibits the proteolytic activity of purified Factor D against substrate Factor B in complex with C3b, blocking production of Bb fragment IC50 of 15 nM.
Danicopan prevented new AP C3 convertase formation, inhibited both AP-mediated hemolysis and C3 fragment deposition on red blood cells from PNH patients.
Danicopan is being developed for the treatment of PNH and other complement-mediated diseases.
Danicopan specifically targets the control point of the complement cascade amplification loop, blocking C3 convertase formation and, significantly reducing production of C3 cleavage products (also known as C3 fragments) and downstream MAC formation.
