Overview

Alexion is a global biopharmaceutical company that discovers, develops and delivers life-changing therapies for patients living with severe, life-threatening and often ultra-rare diseases. With fully integrated scientific, medical, regulatory, manufacturing and commercial capabilities, Alexion is well-positioned to meet the needs of patients today and in the future.

The company's first product, Soliris® (eculizumab), is the first and only therapy approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). PNH is an ultra-rare, debilitating and life-threatening blood disorder defined by the destruction of red blood cells, or hemolysis. In patients with PNH, hemolysis can cause life-threatening thromboses (formation of blood clots), as well as debilitating pain, kidney disease, disabling fatigue, severe anemia and intermittent episodes of dark-colored urine, known as hemoglobinuria.

Soliris is the first in a new class of therapies to inhibit terminal complement, a group of proteins involved in the body's immune response. Extensive clinical trials show that Soliris reduces hemolysis in patients with PNH, leading to an improvement in symptoms and a reduction in major health problems associated with this disease.

Alexion scientists, working closely with leading researchers and physicians worldwide, are investigating eculizumab as a potential treatment for patients with other debilitating and ultra-rare diseases in which complement plays a role. The company also is exploring the potential of a novel antibody as a treatment for chronic lymphocytic leukemia, multiple myeloma and other types of cancer.