Alexion's Soliris® Receives 2009 Prix Galien France for Most Innovative Drug for Rare Disease
CHESHIRE, Conn. & PARIS, Jun 10, 2009 (BUSINESS WIRE) -- Alexion Pharma France and Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN)
today received the 2009 Prix Galien France for Soliris(R)
(eculizumab) in the category of medicines for rare diseases. The award
recognizes the scientific innovation represented by the
complement-inhibition technology of Soliris, and the impact the drug is
having on the lives of patients with paroxysmal nocturnal hemoglobinuria
(PNH), an ultra-rare, debilitating and life-threatening blood disorder.
Soliris is a first-in-class terminal complement inhibitor that
selectively blocks the formation of terminal complement, a component of
the normal immune system. Patients with PNH lack naturally occurring
proteins that ordinarily prevent terminal complement from causing the
red blood cell destruction (hemolysis) that is central to the serious
morbidities and mortality associated with PNH. The French award follows
receipt last year by Alexion of the 2008 Prix Galien USA Award for
Soliris as the Best Biotechnology Product with broad implications for
future biomedical research.
"Alexion's employees in France and throughout Europe are gratified by
this award and by the opportunity we have to improve the lives of
patients with PNH," said Patrice Coissac, Senior Vice President of
Alexion Pharmaceuticals, Inc. and President of Alexion International
Sàrl. "Alexion is committed to the objective that every patient with PNH
who can benefit from Soliris will have access to Soliris, and each day,
we are working with physicians and national healthcare authorities in
Europe toward this goal."
About the Prix Galien
The Prix Galien (http://www.prixgalien.com/)
was established in France in 1970 by French pharmacist Roland Mehl to
recognize and promote significant advances in pharmaceutical research.
The award is among the industry's highest accolades. The French award
committee includes 17 eminent members from the scientific, medical, and
academic communities of France.
Since its debut in France, the Prix Galien has been introduced in other
countries of Europe, as well as in the U.S. and Canada. As in France, a
prominent and independent panel of judges selects the winner, based on
the innovative aspects and therapeutic advantages of the recipients. In
addition, an International Prix Galien is selected from winners of the
country awards every two years.
"We deeply appreciate this honor, which recognizes more than 15 years of
basic and clinical research in the field of complement inhibition," said
Leonard Bell, M.D., Chief Executive Officer of Alexion. "We are building
on the success of Soliris by increasing our understanding of PNH and by
evaluating the promise of complement inhibition for the treatment of
patients with other ultra-rare and life-threatening disorders."
About PNH
Patients with PNH suffer from hemolysis (red blood cell destruction)
which leads to thromboses (blood clots), disabling fatigue, anemia,
impaired quality of life, pulmonary hypertension, shortness of breath,
recurrent pain, kidney disease and intermittent episodes of dark-colored
urine (hemoglobinuria). (1,2) PNH is an ultra-rare blood disorder that
strikes people of all ages, with an average age of onset in the early
30s. (3) Approximately 10 percent of all patients first develop symptoms
at 21 years of age or younger. (1) 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. (4) It is estimated that approximately one-third of
patients with PNH do not survive more than five years from the time of
diagnosis. (4) PNH has been identified more commonly among patients with
disorders of the bone marrow, including aplastic anemia (AA) and
myelodysplastic syndromes (MDS). (5,6,7) 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
(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 paroxysmal
nocturnal hemoglobinuria (PNH), an ultra-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. Soliris is not approved for the
treatment of transplant rejection. 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 U.S. product label for Soliris also includes 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. 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.
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 and kidney diseases, transplant, 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 www.alexionpharma.com.
[ALXN-G]
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 results of clinical trials are not predictive of
safety and efficacy results of Soliris in broader patient populations,
the possibility that third party payers will decide not to reimburse for
use of Soliris at acceptable levels or at all, 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 March 31, 2009. 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. Parker C, Omine M, Richards S, et al. Diagnosis and management of
paroxysmal nocturnal hemoglobinuria. Blood. 2005;106
(12):3699-3709.
2. Hill A, Richards S, Hillmen P. Recent developments in the
understanding and management of paroxysmal nocturnal haemoglobinuria. Br
J Haematol. 2007;137:181-92.
3. 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.
4. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history
of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;
333:1253-1258.
5. 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.
6. 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.
7. 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.
SOURCE: Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Sr. Director
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