Research and Development
Alexion’s R&D programs are focused on discovering and developing highly innovative
therapies with the potential to transform the lives of patients with severe and ultra-rare diseases
who lack adequate treatment options. The company is a world leader in both complement biology and
monoclonal antibody development, and has proven its R&D capabilities through the successful
introduction of Soliris® (eculizumab),
a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is now
approved in more than 35 countries for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and
in the United States and European Union for the treatment of atypical hemolytic uremic syndrome (aHUS)..
Alexion’s R&D efforts are focused primarily in the areas of hematology, nephrology, and neurology. The company is exploring
the potential role of eculizumab in several ultra-rare and life-threatening disorders within these therapeutic areas, as well as
accelerating the development of a broad portfolio of early-stage product candidates through its Translational Medicine group
in Cambridge, Mass. In addition to the eculizumab rare disease programs, Alexion is increasing its focus on the development of
three additional unique drug candidates: TT30, cPMP and a humanized antibody.