Alexion’s development programs are focused on discovering and developing highly innovative
therapies in hematology, nephrology, neurology and metabolic disorders. Our expertise is in
severe and ultra-rare diseases, and our research teams are driven by a mission to transform the
lives of patients who suffer from these debilitating and often life-threatening diseases and lack
Through both investigator-initiated and company-sponsored studies, Alexion is exploring the potential
of eculizumab as well as other novel candidates — including asfotase alfa, TT30, a cPMP replacement therapy and ALXN1007, a humanized antibody — in other severe and ultra-rare disorders.