Alexion Joins NORD, EURORDIS and Other Patient Advocacy Groups in Supporting the Goals of Rare Disease Day 2010
- Global Day of Recognition Underscores Need to Develop, and Provide
Access to, Orphan Therapies -
CHESHIRE, Conn. & SMITHFIELD, R.I., Feb 26, 2010 (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 third annual
Rare Disease Day, February 28, 2010.
Rare Disease Day, established by EURORDIS in 2008, 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. More information on Rare Disease
Day is available at www.rarediseaseday.org.
"Rare Disease Day enables the rare disease community to raise awareness
about the clinical needs and personal struggles faced by these patients
and families. Our goal is to highlight the importance of ongoing
therapeutic breakthroughs and explain the need to facilitate access to
medicines once they become available," said Peter Saltonstall, President
of NORD.
This year's Rare Disease Day theme, "Bridging Patients and Researchers,"
highlights the importance of collaboration between the clinical,
advocacy, and patient communities for the discovery and development of
rare disease treatments. Activities held worldwide marking this year's
Rare Disease Day include educational events, podcasts and social
networking to help patients around the world to share their struggles,
stories and hopes for future treatments.
"The theme of this year's Rare Disease Day is especially meaningful to
Alexion," said Leonard Bell, M.D., Chief Executive Officer of Alexion.
"Through our focus on developing and delivering treatments for patients
with ultra-rare disorders, we know first-hand the possibilities for
transforming patients' lives through medical innovation. We are
continuing to bring Soliris to patients with PNH around the world while
developing treatments for patients with other ultra-rare, debilitating
and life-threatening disorders."
Alexion is the developer of Soliris(R) (eculizumab), the first treatment
approved in the U.S., European Union and other countries as a treatment
for all patients with paroxysmal nocturnal hemoglobinuria (PNH), a
severe, debilitating and life-threatening blood disorder defined by
hemolysis (red blood cell destruction). Among other research programs,
Alexion is currently investigating the potential of Soliris as a
treatment for patients with atypical Hemolytic Uremic Syndrome (aHUS),
an ultra-rare and life-threatening genetic disorder, and acute humoral
rejection in kidney transplant patients. The Company is recognizing Rare
Disease Day with events at its international facilities.
Information about NORD can be found at www.rarediseases.org,
and information about Rare Disease Day activities in the U.S. are
available at www.rarediseaseday.us.
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. (1) More
information on PNH is available at www.pnhsource.com.
About Soliris
Soliris has been approved by the U.S. Food and Drug Administration
(FDA), the European Commission (EC) and healthcare authorities in other
countries as the first treatment for all patients with PNH, an
ultra-rare, debilitating and life-threatening blood disorder defined by
hemolysis, or the destruction of red blood cells. The FDA and EC
reviewed and approved their respective marketing applications for
Soliris under their priority review or accelerated assessment
procedures. In patients with PNH, hemolysis can cause life-threatening
thromboses, recurrent pain, kidney disease, fatigue, impaired quality of
life, anemia, pulmonary hypertension, shortness of breath and
intermittent episodes of dark-colored urine (hemoglobinuria). Soliris is
the only treatment that blocks this hemolysis. Prior to these approvals,
there were no therapies specifically available for the treatment of
patients with 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)
Alexion is committed to the objective that every patient with PNH who
can benefit from Soliris will have access to Soliris. More information
on Soliris is available at www.Soliris.net.
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 commercialize 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, kidney and neurologic diseases, transplant
rejection, cancer and autoimmune disorders. Soliris is Alexion's first
marketed product, approved in the U.S., European Union and other
countries as a treatment for all patients with PNH. Alexion is
evaluating other potential indications for Soliris, and is pursuing
development of other antibody product candidates in early stages of
development. 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 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, 2009, 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) Socié 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. N Engl 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.
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