danicopan + ravulizumab

Danicopan + ravulizumab is a **combination drug regimen** used as add-on therapy for adults with **paroxysmal nocturnal hemoglobinuria (PNH)** who experience clinically significant extravascular hemolysis (cs-EVH) despite standard care with C5 inhibitors. **Danicopan** is an oral, first-in-class, small molecule inhibitor of **complement factor D** (alternative pathway), designed to block C3-mediated extravascular hemolysis. **Ravulizumab** is a humanized monoclonal antibody that inhibits **complement component C5**, thereby blocking terminal complement-mediated intravascular hemolysis. Together, they provide dual blockade at distinct points in the complement cascade to reduce hemolysis and improve anemia, with sustained efficacy and safety demonstrated in phase 3 trials[1][3][5].

Other names

oral complement factor D inhibitor + complement C5 inhibitordanicopan plus ravulizumab