Alexion Pharmaceuticals, formed in 1992, focuses on a strong scientific and product development base to develop novel antibody therapeutics targeting the treatment of patients with a wide array of severe disease states, including autoimmune and cardiovascular disorders, inflammation and cancer. Alexion is dedicated to developing effective and safe treatments for human diseases for which treatment options are either non-existent or inadequate. The company's clinical efforts have focused on treating disease by blocking formation of the terminal components of complement. Complement is an important and natural part of the human immune system that is comprised of a cascade of proteins, one protein leading to formation of the next. The earlier-formed components of the "complement cascade" are generally beneficial, but the later-formed, or terminal, components can have significant and deleterious effects.
Alexion's lead FDA approved product, Soliris (eculizumab), is a humanized monoclonal antibody complement inhibitor. Specifically, eculizumab blocks cleavage of the C5 component of the complement system, thereby preventing the final stages of complement activation. Alexion developed Soliris for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a rare blood disorder characterized by the onset of severe hemolytic anemia, chronic fatigue and intermittent episodes of dark colored urine, known as hemoglobinuria. PNH is a chronic disease where a portion of a patient's oxygen-carrying red blood cells are missing the normally present complement inhibitors and are therefore abnormally fragile and inadvertently destroyed by normal complement activation. PNH has been identified more commonly among patients with disorders of bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS). Underlying bone marrow dysfunction can complicate the clinical course of PNH, contributing to anemia, a greater dependence on transfusion support, an increased risk of infection and life-threatening hemorrhage. Other than Soliris, currently there is no drug specifically available for treatment of patients with PNH.
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