Press Releases
February 27, 2009 at 12:00 AM EST

Alexion Joins NORD and EURORDIS in Supporting the Goals of Rare Disease Day

CHESHIRE, Conn. & PARIS, Feb 27, 2009 (BUSINESS WIRE) -- Alexion Pharmaceuticals, Inc., (Nasdaq: ALXN) has joined the National Organization for Rare Disorders (NORD) and the European Organization for Rare Diseases (EURORDIS) in supporting the goals of the second annual Rare Disease Day, February 28, 2009. Rare Disease Day seeks to increase the visibility of rare diseases, give hope and information to patients, coordinate policy actions in different countries, and ensure equal access to quality care and treatment.

Rare Disease Day was established by EURORDIS in 2008 to focus attention on rare diseases, the challenges encountered by people affected by these conditions, and the importance of research to develop diagnostic tools and treatments. Following the success of the original EURORDIS event in Europe, the 2009 observance, "Patient Care: A Public Affair," will involve hundreds of patient organizations in Europe, the United States, Canada, Australia, and other countries. Activities include social networking to help patients around the world to share their struggles, stories and hopes for future treatments.

"People with rare diseases remain a medically underserved population in every country. Low levels of awareness about rare disorders often result in misdiagnoses or sub-optimal treatment, and diseases that affect small patient populations often have no treatments at all," said Peter Saltonstall, President of NORD. "Rare Disease Day brings together patients and families living with rare diseases to educate the public and policy makers about the need for greater awareness, more research, and better access to diagnosis and treatment."

"It is vitally important to develop treatments for people with rare, severe disorders, and to ensure that patients have access to these therapies once they are available," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "We salute those groups that champion the needs of these patients, including EURORDIS, NORD, the Canadian Organization for Rare Disorders, the Association of Genetic Support of Australasia, and the Leukemia Foundation of Australia. We believe that patients with rare diseases who can benefit from innovative and life-transforming therapies should have access to them. We renew our commitment to this goal each and every day as we work to make Soliris available to patients with PNH. Through international research and development programs, we aim to identify effective therapies for patients with rare disorders, and we are planning to conduct clinical trials of Soliris in at least five other orphan diseases this year."

Alexion is the developer of Soliris(R) (eculizumab), the first treatment approved in the U.S., European Union, Canada and Australia for patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare, debilitating and life-threatening blood disorder.

Information about the global observance of Rare Disease Day can be found at, and information about related activities in the U.S. are available on NORD's website,

About PNH

PNH is a rare blood disorder that strikes people of all ages, with an average age of onset in the early 30s. (1) Approximately 10 percent of all patients first develop symptoms at 21 years of age or younger. (2) PNH develops without warning and can occur in men and women of all races, backgrounds and ages. PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years. (3) The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis. (1,3) PNH has been identified more commonly among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS). (4,5,6) In patients with thrombosis of unknown origin, PNH may be an underlying cause. (2) More information on PNH is available at

About Soliris

Soliris has been approved by the U.S. Food and Drug Administration (March 2007), the European Commission (June 2007), Health Canada (January 2009) and Australia's Therapeutic Goods Administration (February 2009) as the first treatment for all patients with PNH, a rare, debilitating and life-threatening blood disorder defined by hemolysis, or the destruction of red blood cells. All four jurisdictions reviewed and approved their respective marketing applications for Soliris under their priority review or accelerated assessment procedures, and all four have designated Soliris as an orphan drug. Prior to these approvals, there were no therapies specifically available for the treatment of PNH. PNH treatment was limited to symptom management through periodic blood transfusions, non-specific immunosuppressive therapy and, infrequently, bone marrow transplantations -- a procedure that carries its own substantial risks of mortality and morbidity. (2,8) More information on Soliris is available at

Important Safety Information

Soliris is generally well tolerated. The most frequent adverse events observed in clinical studies were headache, nasopharyngitis (a 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 product labels for Soliris in the U.S., Canada and Australia also include a boxed warning: "Soliris increases the risk of meningococcal infections. 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 clinical studies, two out of 196 vaccinated PNH patients treated with Soliris experienced a serious meningococcal infection. In the European Union, the product label for Soliris includes special warning statements that include: "Due to its mechanism of action, the use of Soliris increases the patient's susceptibility to meningococcal infections (Neisseria meningitidis)." "To reduce the risk of infection, all patients must be vaccinated at least 2 weeks prior to receiving the first dose of Soliris and must be revaccinated according to current medical guidelines for vaccine use." And, "All patients should be monitored for early signs of meningococcal infections, evaluated immediately if infection is suspected, and treated with antibiotics if necessary. Patients should be informed of these signs and symptoms and steps taken to seek medical care immediately."

Prior to beginning Soliris therapy, all patients and their prescribing physicians are enrolled in the Soliris Safety 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.

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company working to develop and deliver life-changing drug therapies for patients with serious and life-threatening medical conditions. Alexion is engaged in the discovery, development and commercialization of therapeutic products aimed at treating patients with a wide array of severe disease states, including hematologic diseases, cancer, and autoimmune disorders. Soliris is Alexion's first marketed product, approved in the U.S. and Europe in 2007, and Canada and Australia in 2009. Alexion is evaluating other potential indications for Soliris as well as other formulations of eculizumab for additional clinical indications, and is pursuing development of other antibody product candidates in early stages of development. This press release and further information about Alexion Pharmaceuticals, Inc. can be found at:


Safe Harbor Statement

This news release contains forward-looking statements, including statements related to potential health and medical benefits from Soliris. 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, the possibility that initial results of commercialization are not predictive of future rates of adoption of Soliris, 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 Annual Report on Form 10-K for the period ended December 31, 2008, 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) Socie G, Mary J Yves, de Gramont A, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. Lancet. 1996: 348:573-577.

(2) Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106 (12):3699-3709.

(3) Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. NEngl J Med. 1995; 333:1253-1258.

(4) Wang H, Chuhjo T, Yasue S, Omine M, Naka S. Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood. 2002;100 (12):3897-3902.

(5) Iwanga M, Furukawa K, Amenomori T, et al. Paroxysmal nocturnal haemoglobinuria clones in patients with myelodysplastic syndromes. Br J Haematol. 1998;102 (2):465-474.

(6) Maciejewski JP, Risitano AM, Sloand EM, et al. Relationship between bone marrow failure syndromes and the presence of glycophosphatidyl inositol-anchored protein-deficient clones. Br J Haematol. 2001;115:1015-1022.

(7) Hill A, Richards S, Hillmen P. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2007;137 (3):181-192.

(8) Hill A, Richards S, Hillmen P. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2007;137 (3):181-192.

SOURCE: Alexion Pharmaceuticals, Inc.

Alexion Pharmaceuticals, Inc.
Irving Adler, 203-272-8210
Sr. Director, Corporate Communications
Makovsky + Company
Mark Marmur, 212-508-9670
Rx Communications
Rhonda Chiger, 917-322-2569

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