Alexion cheers success of anemia therapy

Paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening form of hemolytic anemia, is an acquired genetic blood disorder characterized by destruction of red blood cells by the body's complement system (a component of the immune system). Patients with PNH lack naturally-occurring complement inhibitors on the surface of their red blood cells that normally prevent red blood cell destruction.

Soliris (eculizumab) is a humanized monoclonal antibody drug designed to selectively block terminal complement activation, thereby preventing destruction of red blood cells by complement in patients with PNH. There currently is no therapy specifically available for the treatment of PNH.

Alexion said that data from the study showed that Soliris may provide significant clinical benefit to a broader, more diverse population of PNH patients than previously observed.

The pre-specified primary endpoints of the trial were safety and a reduction in intravascular hemolysis (destruction of red blood cells).

“PNH patients treated with eculizumab experienced statistically and clinically important improvements in anemia and quality of life over a prolonged period of time in a controlled clinical setting on a global basis,” said Dr Robert Brodsky, director, Division of Hematology, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University School of Medicine.