Research shows that danicopan added to ravulizumab or eculizumab can have long-term efficacy in controlling paroxysmal nocturnal hemoglobinuria (PNH).
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder characterized by abnormal destruction of red blood cells during sleep. Previous research has shown that monoclonal antibodies ravulizumab and eculizumab can improve outcomes and survival. However, some patients on those treatments remain anemic due to extravascular hemolysis.
PNH results from a defective gene, which causes a malfunction in the complement system, part of the immune system. Danicopan inhibits a protein called complement factor D, which prevents red blood cells from being marked for destruction.