Soliris is the first therapy approved for paroxysmal nocturnal hemoglobinuria (PNH), a rare, disabling and life-threatening blood disorder defined by chronic red blood cell destruction, or hemolysis, the company said. Soliris is indicated for the treatment of patients with PNH to reduce hemolysis.
Hemolysis can cause severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark colored urine, kidney disease, impaired quality of life and blood clots.
Patients with PNH are missing a specific protein that normally protects red blood cells from destruction by a component of the immune system called terminal complement. Soliris, the first complement inhibitor approved in the US for the treatment of any disease, prevents hemolysis by selectively blocking terminal complement.
Leonard Bell, CEO of Alexion, said: “Soliris brings real hope to people who live daily with the devastating effects of PNH. With the approval of Soliris, we now have a therapy that dramatically improves the lives of patients suffering from this disease.”