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, neurology and metabolic disorders.
In addition to the eculizumab development programs in several ultra-rare and life-threatening disorders, Alexion is increasing
its focus on the development of four unique drug candidates: asfotase alfa, TT30, cPMP replacement therapy, and a humanized antibody called ALXN1007.