NCIt definition : An orally bioavailable inhibitor of complement factor D (FD; CFD), a serine protease
that cleaves complement factor B, with potential complement system inhibiting activity.
Upon administration, danicopan targets, binds to and blocks the activity of FD, and
thereby inhibits cleavage of complement factor B into Ba and Bb in the alternative
pathway of the complement cascade. This inhibits FD-mediated signaling and activation
of the alternative complement pathway (ACP), blocks complement-mediated hemolysis
in paroxysmal nocturnal hemoglobinuria (PNH) and prevents ACP-induced tissue damage.
FD plays a key role in the activation of the ACP.;
UNII : JM8C1SFX0U;
InChIKey : PIBARDGJJAGJAJ-NQIIRXRSSA-N;
CAS number : 1903768-17-1;
Molecule name : ACH 4471; ACH-0144471; ACH-4471; ALXN-2040; ALXN 2040;