Alexion's Soliris® (Eculizumab) Receives Marketing Approval in Japan for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
First Therapy Approved for Patients with PNH, a Rare and
Life-Threatening Blood Disease
Soliris is a First-in-Class Complement Inhibitor Antibody
CHESHIRE, Conn. & LAUSANNE, Switzerland, Apr 16, 2010 (BUSINESS WIRE) -- Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) and Alexion Pharma
International Sàrl today announced that Japan's Ministry of Health,
Labour and Welfare (MHLW) has approved the Company's New Drug
Application (NDA) for the use of Soliris(R) (eculizumab) as a treatment
for patients in Japan with paroxysmal nocturnal hemoglobinuria (PNH).
PNH is an ultra-rare, debilitating and life-threatening blood disorder
defined by chronic red blood cell destruction, or hemolysis. Soliris, a
first-in-class terminal complement inhibitor, is the first therapy
approved in Japan for the treatment of patients with PNH. Soliris
received orphan drug designation from the MHLW in 2009 and was approved
under the Ministry's priority review process.
"The speedy approval of the Soliris NDA in Japan underscores the
severity of PNH in Japanese patients and the significant clinical impact
that this treatment provides to patients living with PNH," said Leonard
Bell, M.D., Chief Executive Officer of Alexion. "This regulatory
approval marks another important step in our global commitment to the
objective of providing access to Soliris to all patients who can benefit
from it. We now look forward to working closely with the healthcare
authorities in Japan to make Soliris available to patients as rapidly as
possible."
Soliris was approved as a treatment for patients with PNH by the U.S.
Food and Drug Administration and the European Commission in 2007, and
has since received similar approvals from the healthcare authorities in
other countries, including Australia, Korea and Canada. Governments and
private insurance companies have recognized the breakthrough innovation
of Soliris and are now providing patients with broad access to Soliris
in the United States, the largest European nations, and additional
countries around the world. With the approval of the NDA, Alexion is
working with the MHLW to facilitate patient access to Soliris. The
Company continues to anticipate a commercial launch of Soliris in Japan
by the end of 2010.
"PNH is very rare, but has a devastating impact on many of the patients
that it affects. After decades of ground-breaking basic scientific
research in Japan regarding PNH, it is particularly noteworthy that
clinical studies, including the AEGIS trial in Japan, have shown that
Soliris markedly reduces hemolysis, the underlying cause of the serious
illness and shortened life-span associated with PNH," said Yuzuru
Kanakura, M.D., Ph.D., Professor of Hematology and Oncology at Osaka
University Hospital in Suita, Japan, and lead investigator of the AEGIS
study. "Japanese patients with PNH will soon have access to the same
life-changing medical benefits of Soliris that are available to patients
in other nations."
The AEGIS Study in Japan
Alexion's NDA for Soliris included data from AEGIS, an open-label
registration study examining Soliris as a treatment for Japanese
patients with PNH, (1) as well as data from the previously reported
SHEPHERD and TRIUMPH (2,3) PNH registration trials, which were conducted
in North America, Europe, and Australia. AEGIS was conducted during 2008
and included 29 patients at nine institutions throughout Japan.
In December 2008, Alexion reported positive results from AEGIS. (4) 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.
AEGIS Extension Study - Additional Positive Data Regarding Hemolysis
and Chronic Kidney Disease
In December 2009, Alexion announced positive data from the 26-week
extension of the AEGIS study. (5) Results showed that there was a
sustained reduction in intravascular hemolysis, as measured by lactate
dehydrogenase (LDH), through the 38 weeks of treatment. LDH decreased
87% from a median of 1,814 U/L at baseline to a median of 232 U/L at 38
weeks of treatment (p<0.001).
A significant improvement in chronic kidney disease (CKD) stage was also
seen in Japanese patients on long-term Soliris treatment. Two-thirds
(19/29) of patients enrolled in the 12-week AEGIS study demonstrated
evidence of CKD at baseline prior to eculizumab. Soliris treatment
significantly increased the likelihood of improvement in CKD in Japanese
patients: at week 38, 53% (9/17) of patients with CKD at baseline
demonstrated improvement.
About PNH
PNH is a rare blood disorder that strikes people of all ages, with an
average age of onset in the early 30s. (6) Approximately 10 percent of
all patients first develop symptoms at 21 years of age or younger. (7)
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. (8) It is estimated
that approximately one-third of patients with PNH do not survive more
than five years from the time of diagnosis. (8) PNH has been identified
more commonly among patients with disorders of the bone marrow,
including aplastic anemia (AA) and myelodysplastic syndromes (MDS).
(9,10,11) In patients with thrombosis of unknown origin, PNH may be an
underlying cause. (6) More information on PNH is available at www.pnhsource.com.
About Soliris
Soliris (eculizumab) is a first-in-class terminal complement inhibitor
developed from the laboratory through regulatory approval by Alexion.
Soliris has been approved by the healthcare authorities in the U.S.,
European Union and other countries as the first treatment for patients
with PNH, a rare, debilitating and life-threatening blood disorder
defined by hemolysis, or the destruction of red blood cells. Prior to
these approvals, there was no therapy specifically available for the
treatment of PNH.
Outside of Japan, patients with PNH in more than 20 countries now have
access to Soliris therapy through national or private healthcare
providers. As the first terminal complement inhibitor to be approved in
countries around the world, Soliris represents a long-sought
breakthrough in medical innovation. Alexion's innovative approach to
complement inhibition has received some of the pharmaceutical industry's
highest honors: the 2008 Prix Galien USA Award for Best Biotechnology
Product with broad implications for future biomedical research, and the
2009 Prix Galien France Award in the category of Drugs for Rare
Diseases. 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. Meningococcal
infection may become rapidly life-threatening or fatal if not recognized
and treated early. 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, other
inflammatory disorders, and cancer. Soliris is Alexion's first marketed
product. 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
published reports or clinical trials are not predictive of safety and
efficacy results of Soliris in broader patient populations, the risk
that clinical trials may not be completed successfully, 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, 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, 2010, 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) 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.
(5) Kanakura Y, Ohyashiki K, Shichishima T et al. Chronic Renal
Insufficiency in Japanese Patients with Paroxysmal Nocturnal
Hemoglobinuria (PNH): Improvement with Eculizumab Treatment in the
Long-Term Follow-up of the AEGIS Study. Blood 2009;114:A1980.
(6) 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.
(7) Parker C, Omine M, Richards S, et al. Diagnosis and management of
paroxysmal nocturnal hemoglobinuria. Blood. 2005;106 (12):3699-3709.
(8) Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural
history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;
333:1253-1258.
(9) 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.
(10) 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.
(11) 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
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