danicopan + eculizumab

Danicopan + eculizumab is a combination therapy of two drugs used to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular hemolysis (cs-EVH) while on complement C5 inhibitor therapy. **Danicopan** is a first-in-class, oral, small molecule inhibitor of complement factor D, which blocks the formation of the C3 convertase in the alternative complement pathway, thereby preventing both intravascular and extravascular hemolysis. **Eculizumab** is a monoclonal antibody that inhibits complement component C5, blocking terminal complement-mediated intravascular hemolysis. The combination aims to optimize control of both intravascular and extravascular hemolysis, with data from phase 3 studies demonstrating significant increases in hemoglobin, reduced need for transfusion, and sustained efficacy and safety over long-term follow-up. The therapy is intended for adults with PNH who remain anemic due to cs-EVH despite ongoing treatment with eculizumab[1][2][3][5].

Other names

danicopan + eculizumab