In patients with PNH, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, nasopharyngitis (runny nose), back pain and nausea. Soliris treatment of patients with PNH should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established. In patients with aHUS, the most frequently reported adverse events observed with Soliris treatment in clinical studies were hypertension, upper respiratory tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract infection, and leukopenia. Please see full prescribing information for Soliris, including boxed WARNING regarding risk of serious meningococcal infection.
Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on serving patients with severe and ultra-rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris ® (eculizumab) as a treatment for patients with PNH and aHUS, two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is currently approved in more than 40 countries for the treatment of PNH, and in the United States and the European Union for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris and is developing four other highly innovative biotechnology product candidates, which are being investigated across eight severe and ultra-rare disorders beyond PNH and aHUS. This press release and further information about Alexion Pharmaceuticals, Inc. can be found at: www.alexionpharma.com.
[ALXN-G]Safe Harbor Statement This news release contains forward-looking statements, including statements related to anticipated potential health and medical benefits of Soliris® (eculizumab) for the treatment of patients with PNH and aHUS. Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ from those expected, including for example, decisions of regulatory authorities regarding marketing approval or material limitations on the marketing of Soliris for its current or potential new indications, and a variety of other risks set forth from time to time in Alexion's filings with the Securities and Exchange Commission, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended September 30, 2012, and in Alexion's other filings with the Securities and Exchange Commission. Alexion does not intend to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.
|1.||Hill A, Kelly R, Kulasekararaj A, et al. Eculizumab in paroxysmal nocturnal hemoglobinuria (PNH): a report of all 153 patients treated in the UK. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 10, 2012: Abstract 3472.|
|2.||Szer J, Muus P, Roth A, et al. Long-term safety of sustained eculizumab treatment in patients with paroxysmal nocturnal hemoglobinuria. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 8, 2012: Abstract 1260.|
Socié G, Schrezenmeier H, Muus P, et al. Eculizumab protects against TE and prolongs survival in patients with paroxysmal nocturnal hemoglobinuria: an International PNH Registry study. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 10, 2012: Abstract 3480.
|4.||Lee JW, Jang JH, Kim JS, et al. Risk of thromboembolism in patients with paroxysmal nocturnal hemoglobinuria presenting with both clinical symptoms and elevated hemolysis. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 8, 2012: Abstract 1273.|
|5.||Nishimura J, Yamamoto M, Hayashi S, et al. A rare genetic polymorphism in C5 confers poor response to the anti-C5 monoclonal antibody eculizumab by nine Japanese patients with PNH. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 10, 2012: Abstract 3197.|
|6.||Licht C, Muus P, Legendre C, et al. Eculizumab (ECU) safety and efficacy in atypical hemolytic uremic syndrome (aHUS) patients with long disease duration and chronic kidney disease: 2-year results. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 8, 2012: Abstract 985.|
|7.||Greenbaum L, Legendre C, Babu S, et al. Eculizumab (ECU) in atypical hemolytic uremic syndrome (aHUS) patients with progressing thrombotic microangiopathy (TMA): 2-year data. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 9, 2012: Abstract 2084.|
Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333:1253-1258.
Kelly RJ, Hill A, Arnold LM, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011;117(25):6786-6792.
Dr. Anita Hill receives research support from Alexion Pharmaceuticals, Inc. and has served on advisory boards for the company.
|11.||Dr. Jeffrey Szer has no relevant conflicts of interest to disclose.|
|12.||Dr. Gerard Socié has no relevant conflicts of interest to disclose.|
|13.||Movalia MK, Illingworth A. Distribution of PNH clone sizes within high risk diagnostic categories among 481 PNH positive patients identified by high sensitivity flow cytometry. Presented at the 54th Annual Meeting of the American Society of Hematology (ASH), Atlanta, GA, December 8, 2012: Abstract 1271.|
Two-year data show long-term benefits of chronic Soliris ® therapy in patients with aHUS (press release). Alexion Pharmaceuticals, Inc.: November 3, 2012. http://news.alexionpharma.com/press-release/product-news/two-year-data-show-long-term-benefits-chronic-soliris-therapy-patients-ah.
|15.||Dr. Larry Greenbaum receives research support from Alexion Pharmaceuticals, Inc. and is a consultant to the company.|
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