The complement system is a robust and complex branch of the body’s natural immune system. When the complement system detects foreign particles in the body, it triggers a cascade of complement protein activation, resulting in destruction or removal of the invading particles. Normally, our body’s cells and tissues are protected from complement activation by naturally occurring complement regulatory proteins anchored to the surface of cells. While complement activation is essential to maintaining health, the complement system can become uncontrolled when excessive complement activation occurs or when these naturally occurring complement regulatory proteins are missing on the surface of cells. Uncontrolled complement activation can play a role in numerous chronic and life-threatening diseases.
The complement system offers a rich group of targets for inhibiting the inflammatory process. Alexion is a world leader in complement inhibition and was the first company to discover, develop and commercialize a terminal complement inhibitor, Soliris® (eculizumab). Soliris is approved for the treatment of patients with PNH, a progressive and life-threatening disease characterized by excessive destruction of red blood cells, and for the treatment of patients with aHUS, an ultra-rare, life-threatening, genetic disease that progressively damages vital organs. . Soliris has received two of the industry’s highest honors for innovation: the 2008 Prix Galien USA Award for Best Biotechnology Agent and the 2009 Prix Galien France Award in the category of medicines for rare diseases.
Today, we are actively investigating the potential role of our terminal complement inhibitor eculizumab in the treatment of other ultra-rare diseases in which uncontrolled complement activity is life-threatening for patients. We are also evaluating a broad portfolio of antibody and protein regulators of the complement inflammatory pathway.