2011 Revenue Guidance Revised Upward
Both the US and EU filings include the positive data from the two 26-week Phase 2 studies of Soliris as a treatment for adult and adolescent patients with aHUS. Preliminary data from these two studies were presented at the
"The US and EU regulatory submissions put us one step closer toward accomplishing our goal to transform the lives of patients suffering with aHUS," said
Updated 2011 Revenue Guidance
Alexion is updating its 2011 revenue guidance, from the previously announced range of
The upward revision in revenue guidance takes into account continued global growth of Soliris for PNH, and the potential for an earlier than previously expected US launch of Soliris for aHUS. The earlier launch could occur if the US regulatory submission is accepted for priority review by the
aHUS is a chronic, ultra-rare disease characterized by thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body, causing a reduction in platelet count and life-threatening damage to the kidney, brain, heart and other vital organs.4-6 Approximately 60 percent of patients with aHUS require dialysis or a kidney transplant or die within a year of diagnosis.7 The majority of patients with aHUS who receive a kidney transplant experience severe complications of the disease, and more than 90 percent of these patients experience failure of the donor kidney.8
aHUS is a progressive disease caused by life-long uncontrolled activation of the complement system due to deficiencies in complement regulatory genes. With genetic deficiency of naturally occurring complement inhibitors, patients experience chronic uncontrolled activation of the complement system, causing ongoing inflammation and blood clots in vital organs.9,10 In patients with aHUS, uncontrolled complement activation results in an ongoing risk of sudden and catastrophic life-threatening complications. Currently, mutations have been identified in at least ten different genes; however, in approximately one-half of patients diagnosed with aHUS, the specific genetic deficiency cannot currently be identified.
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris has been approved in the U.S.,
Important Safety Information
Soliris is generally well tolerated in patients with PNH. The most frequent adverse events observed in clinical studies of patients with PNH were headache, nasopharyngitis (runny nose), back pain and nausea. Treatment with Soliris should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established.
The U.S. product label for Soliris also includes a boxed warning: "Soliris increases the risk of meningococcal infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Vaccinate patients with a meningococcal vaccine at least two weeks prior to receiving the first dose of Soliris; revaccinate according to current medical guidelines for vaccine use. Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary." During PNH clinical studies, two out of 196 vaccinated PNH patients treated with Soliris experienced a serious meningococcal infection. Prior to beginning Soliris therapy, all patients and their prescribing physicians are encouraged to enroll in the PNH Registry, which is part of a special risk-management program that involves initial and continuing education and long-term monitoring for detection of new safety findings.
Safe Harbor Statement
This news release contains forward-looking statements, including statements related to anticipated clinical development milestones and potential health and medical benefits of Soliris® (eculizumab) for the potential treatment of patients with 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
(1) Abstract 1338 entitled "Safety and Efficacy of Eculizumab in aHUS Patients Resistant to Plasma Therapy: Interim Analysis from a Phase II Trial," presented in an oral presentation at the
(2) Alexion Pharmaceuticals Corporate Website. Press release available from URL: http://www.alxn.com/News/article.aspx?relid=532082.
(3) Abstract 157 entitled "Safety and Efficacy of Eculizumab in aHUS Patients on Chronic Plasma Therapy: Interim Analysis of a Phase II Trial," presented in a poster presentation at the
(5) Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin Nephrol Hypertens 2010 May;19(3):242-7.
(6) Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int 2006 Jul;70(1):16-23.
(7) Loirat C, Noris M, Fremeaux-Bacchi V. Complement and the atypical hemolytic uremic syndrome in children. Pediatr Nephrol. 2008;23(11):1957-1972.
(8) Bresin E, Daina E, Noris M, Castelletti F, Stefanov R, Hill P, et al. Outcome of renal transplantation in patients with non-Shiga toxin-associated hemolytic uremic syndrome: prognostic significance of genetic background. Clin J Am Soc Nephrol 2006 Jan;1(1):88-99.
(9) Hosler GA, Cusumano AM, Hutchins GM. Thrombotic thrombocytopenic purpura and hemolytic uremic syndrome are distinct pathologic entities. A review of 56 autopsy cases. Arch Pathol Lab Med 2003 Jul;127(7):834-9.
(10) Ståhl A, Vaziri-Sani F, Heinen S, Kristoffersson A-C, Gydell K-H, Raafat R, Gutierrez A, Beringer O, Zipfel PF, and Karpman D. Factor H dysfunction in patients with atypical hemolytic uremic syndrome contributes to complement deposition on platelets and their activation. Blood. 2008;111:5307-5315.
Senior Director, Corporate Communications and Public Policy
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