New Data Detailing the Devastating Nature of Hypophosphatasia (HPP) and Treatment Effect of Asfotase Alfa in Infants, Young Children and Juveniles with HPP Presented at the Pediatric Academic Societies Meeting | Alexion Pharmaceuticals, Inc

CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) today announced that researchers have presented new data from a large, retrospective, natural history study of patients with severe perinatal and infantile hypophosphatasia (HPP), an inherited, rare metabolic disorder that can lead to progressive damage to multiple vital organs, destruction and deformity of bones, and death. The natural history study details the devastating nature of the disease, with 73% mortality reported at five years. Additionally, early and sustained improvements were observed in infants, children and juveniles receiving asfotase alfa, an investigational therapy for the treatment of HPP, in the open-label extension phase of two on-going Phase 2 clinical studies. The data were presented in poster sessions at the joint meeting of the Pediatric Academic Societies (PAS) and the Asian Society for Pediatric Research in Vancouver, B.C., Canada. - See more at: http://news.alexionpharma.com/press-release/product-news/new-data-detailing-devastating-nature-hypophosphatasia-hpp-and-treatment-#sthash.IJMGbVi2.dpuf



New Data Detailing the Devastating Nature of Hypophosphatasia (HPP) and Treatment Effect of Asfotase Alfa in Infants, Young Children and Juveniles with HPP Presented at the Pediatric Academic Societies Meeting | Alexion Pharmaceuticals, Inc

FDA Approves Conversion of Soliris® (eculizumab) Accelerated Approval in aHUS to Regular Approval for the Treatment of Patients with aHUS | Alexion Pharmaceuticals, Inc

CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) today announced that the U.S. Food and Drug Administration (FDA) has approved the Company’s supplemental Biologics License Application (sBLA) providing regular approval for Soliris® (eculizumab) for the treatment of adult and pediatric patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). This update reflects Alexion’s fulfillment of post-marketing requirements, including the submission of confirmatory data from two additional prospective clinical trials, including one in pediatric patients with aHUS. The revised label now specifies important longer-term clinical benefit associated with chronic and sustained Soliris treatment with inclusion of results with two years of ongoing treatment in aHUS patients. The updated label also includes data on the use of Soliris treatment prior to use of supportive care with either plasma exchange or plasma in prospective clinical trials. - See more at: http://news.alexionpharma.com/press-release/product-news/fda-approves-conversion-soliris%C2%A0eculizumab-accelerated-approval-ahus-regu#sthash.5Fg0dZcN.dpuf



FDA Approves Conversion of Soliris® (eculizumab) Accelerated Approval in aHUS to Regular Approval for the Treatment of Patients with aHUS | Alexion Pharmaceuticals, Inc