Alexion Submits NDA in Japan for Soliris® (eculizumab) as a Treatment for Patients with PNH
CHESHIRE, Conn., Apr 01, 2009 (BUSINESS WIRE) -- Alexion Pharmaceuticals, Inc., (Nasdaq: ALXN) today announced that it
has submitted a New Drug Application (NDA) for Soliris? (eculizumab) as
a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH)
to Japan's Pharmaceuticals and Medical Devices Agency (PMDA). PNH is a
rare, debilitating and life-threatening blood disorder defined by
chronic red blood cell destruction, or hemolysis.
AEGIS Study in Japan
The NDA included data from AEGIS, an open-label registration study
examining Soliris as a treatment for Japanese patients with PNH. (1)
AEGIS was conducted to confirm the safety and efficacy of Soliris in
Japanese patients with PNH relative to the previously reported SHEPHERD
and TRIUMPH (2,3) Phase III Soliris trials, which were conducted in the
United States, Europe, and Australia. AEGIS was conducted during 2008
and included 29 patients at nine institutions throughout Japan. Patients
in AEGIS were dosed in accordance with the Soliris product labels
approved in the United States and European Union in 2007. (1)
On December 8, 2008, Alexion reported positive results from AEGIS. The
prespecified primary efficacy endpoint of change in hemolysis was
achieved with an 86 percent reduction (P<0.001). Key secondary endpoints
including improvement in fatigue (P<0.001) and reduction in transfusions
(P<0.001) were also achieved. The drug appeared to be safe and well
tolerated in study patients; most adverse events were mild to moderate
in severity, with the most frequent being headache (52 percent),
nasopharyngitis (41 percent), and nausea (21 percent). No serious
adverse events related to study drug were reported following treatment
with Soliris. (1)
Orphan Drug Designation
In January 2009, Soliris was designated as an orphan drug by Japan's
Ministry of Health, Labour and Welfare. As a result of the designation,
the NDA will receive priority review from the PMDA, and the drug would
have 10 years of market exclusivity as a treatment for patients with
paroxysmal nocturnal hemoglobinuria (PNH) in Japan upon approval.
"This NDA submission is an important step toward improving the lives of
patients suffering from PNH in Japan," said Leonard Bell, M.D., Chief
Executive Officer of Alexion. "Given Japan's long-standing leadership in
PNH research, the debilitating nature of this rare and serious disease,
and the markedly positive results of the AEGIS study, we know that the
Japanese medical community is eager to provide Soliris to patients as
quickly as the regulatory process can allow."
Alexion has begun to establish its commercial organization in Japan in
anticipation of a commercial launch of Soliris in that country in 2010.
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) It is estimated
that approximately one-third of patients with PNH do not survive more
than five years from the time of diagnosis. (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) 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 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. (5,10) 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 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: 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
and the timing of regulatory and commercial milestones for Soliris in
Japan. 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-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) Kanakura Y, Ohyashiki K, Shichishima T, et al. Safety and efficacy
of the terminal complement inhibitor eculizumab in Japanese patients
with paroxysmal nocturnal hemoglobinuria: AEGIS phase II clinical study
results [abstract]. Blood. 2008;112:A3438.
(2) Brodsky RA, Young, NS, Antonioli E, et al. Multicenter phase 3 study
of the complement inhibitor eculizumab for the treatment of patients
with paroxysmal nocturnal hemoglobinuria. Blood.
2008;111:1840-1847.
(3) Hillmen P, Young NS, Schubert J, et al. The complement inhibitor
eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med.
2006;355:1233-1243.
(4) Soci? G, Mary J Yves, de Gramont A, et al. Paroxysmal nocturnal
haemoglobinuria: long-term follow-up and prognostic factors. Lancet.
1996: 348:573-577.
(5) Parker C, Omine M, Richards S, et al. Diagnosis and management of
paroxysmal nocturnal hemoglobinuria. Blood. 2005;106
(12):3699-3709.
(6) Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural
history of paroxysmal nocturnal hemoglobinuria. N Engl J Med.
1995; 333:1253-1258.
(7) 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.
(8) 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.
(9) 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.
(10) 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, 203-271-8210
Sr. Director Corporate Communications
or
Makovsky & Company
Media:
Mark Marmur, 212-508-9670
or
Rx Communications
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Rhonda Chiger, 917-322-2569
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