Alexion's Soliris® (eculizumab) Receives Marketing Approval in Canada for All Patients with PNH
CHESHIRE, Conn., Jan 29, 2009 (BUSINESS WIRE) -- Alexion Pharmaceuticals, Inc., (Nasdaq: ALXN) today announced that
Canada's national healthcare regulatory agency, Health Canada, has
approved the use of Soliris(R) (eculizumab) for the treatment of all
patients in Canada with paroxysmal nocturnal hemoglobinuria (PNH), a
rare, debilitating and life-threatening blood disorder defined by
chronic red blood cell destruction, or hemolysis. Soliris is the first
therapy approved in Canada for the treatment of PNH.
"Soliris has had a life-changing impact on patients with PNH and
represents the only effective and safe drug therapy available for their
disease," said Dr. Loree M. Larratt, M.D., Department of Medicine,
Divisional Director Clinical Hematology, University of Alberta at
Edmonton, and one of the researchers who participated in clinical trials
of Soliris. "Hemolysis underlies the significant morbidities and
mortality of PNH, and Soliris reduced hemolysis in every patient treated
in clinical studies."
Soliris was approved under Priority Review by Health Canada's Biologics
and Genetic Therapies Directorate (BGTD). The marketing application
submitted to the BGTD included safety and efficacy data from three
multi-national clinical studies: TRIUMPH, a placebo-controlled 26-week
Phase 3 study involving 87 PNH patients, (1) SHEPHERD, an open-label
52-week Phase 3 trial involving 97 PNH patients, (2) and E05-001, a long
term extension study. (3) Soliris was approved by the United States Food
and Drug Administration and the European Commission in 2007 using data
from the same studies and is currently being used to treat patients with
PNH in the U.S. and more than 15 additional countries.
"This approval paves the way for patients with PNH across Canada to
begin receiving the clinical benefits of Soliris," said Leonard Bell,
M.D., Chief Executive Officer of Alexion. "We are currently developing
our organization in Canada and we will now work with Canada's public and
private healthcare organizations to provide access to Soliris,
patient-by-patient, as rapidly as possible."
"We are very excited that Health Canada has been able to move quickly to
approve Soliris. We have been hearing from Canadian patients affected by
PNH who have been waiting for access to this innovative drug," said
Durhane Wong-Rieger, Ph.D., President of the Canadian Organization for
Rare Disorders. "We call upon the private, provincial and federal drug
plans to act without delay to provide this life-saving therapy to
patients as soon as possible."
About PNH
PNH is a rare blood disorder that strikes people of all ages, with an
average age of onset in the early 30s. (4) Approximately 10 percent of
all patients first develop symptoms at 21 years of age or younger. (5)
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. (6) The estimated
median survival for PNH patients is between 10 and 15 years from the
time of diagnosis. (4,6) PNH has been identified more commonly among
patients with disorders of the bone marrow, including aplastic anemia
(AA) and myelodysplastic syndromes (MDS). (7,8,9) In patients with
thrombosis of unknown origin, PNH may be an underlying cause. (5)
Prior to approval of Soliris in the U.S. and European Union, 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 considerable
mortality risk. (5,10)
About Soliris
Soliris was approved in March 2007 by the U.S. Food and Drug
Administration (FDA) as the first treatment for PNH, a rare,
debilitating and life-threatening blood disorder defined by hemolysis,
or the destruction of red blood cells. In June 2007, the European
Commission (EC) also approved the use of Soliris for the treatment of
patients with PNH. Soliris is the first therapy approved in Europe for
the treatment of PNH and was the first medicinal product to receive EC
approval under the EMEA Accelerated Assessment Procedure.
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 Canadian 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 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. In
March 2007, the FDA granted marketing approval for Alexion's first
product, Soliris, for all patients with PNH, and Alexion began
commercial sale of Soliris in the U.S. during April 2007. In June 2007,
the EC granted marketing approval for Soliris in the European Union for
all patients with PNH. 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.alexionpharm.com.
[ALXN-G]
Safe Harbor Statement
This news release contains forward-looking statements, including
statements related to potential health and medical benefits from Soliris
and the timing of regulatory and commercial milestones for Soliris in
Canada. 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, delays in
arranging satisfactory manufacturing capability and establishing
commercial infrastructure, delays in developing or adverse changes in
commercial relationships, the possibility that results of clinical
trials are not predictive of safety and efficacy results of Soliris in
broader patient populations, the possibility that initial results of
commercialization are not predictive of future rates of adoption of
Soliris, the risk that third parties won't agree to license any
necessary intellectual property to Alexion on reasonable terms or at
all, the risk that third party payors will not reimburse for the use of
Soliris at acceptable rates or at all, the risk that estimates regarding
the number of PNH patients are inaccurate 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, 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.
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(7) Wang H, Chuhjo T, Yasue S, Omine M, Naka S. Clinical significance of
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(8) Iwanga M, Furukawa K, Amenomori T, et al. Paroxysmal nocturnal
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(9) Maciejewski JP, Risitano AM, Sloand EM, et al. Relationship between
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SOURCE: Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Irving Adler, 203-272-8210
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