Soliris® Receives Orphan Drug Designation in Japan
CHESHIRE, Conn., Jan 12, 2009 (BUSINESS WIRE) --
Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today reported that
Soliris(R) (eculizumab), its first-in-class complement inhibitor, has
been designated as an orphan drug by the Ministry of Health, Labour and
Welfare of Japan. As a result of the designation, a New Drug Application
(NDA) for Soliris would receive priority review from the Japanese
regulatory authorities once it is submitted, and the drug would have 10
years of market exclusivity as a treatment for patients with paroxysmal
nocturnal hemoglobinuria (PNH) in Japan.
Soliris was previously granted orphan drug status in the United States
and European Union, and has been in use in the U.S. and European
countries following regulatory approvals in 2007. AEGIS, an open-label
registration study examining Soliris as a treatment for Japanese
patients with PNH, was conducted during 2008, and on December 8, 2008
Alexion reported positive results from AEGIS. Alexion expects to include
data from the AEGIS study in an NDA to be submitted in Japan in 2009.
The period of market exclusivity under the orphan drug designation would
commence upon approval of that application. The Company has begun to
establish its commercial organization in Japan in anticipation of a
commercial launch of Soliris in that country in 2010.
"The orphan drug designation reflects the therapeutic value of Soliris
as a treatment for Japanese patients suffering with PNH, who currently
lack an effective drug therapy for their disease," said Dr. Mitsuhiro
Omine, Visiting Professor, Showa University Division of Hematology,
Internal Medicine, Japan.
"Japanese scientists conducted much of the early research in PNH," said
Leonard Bell, M.D., Chief Executive Officer of Alexion. "In light of the
long-standing awareness of PNH in Japan, and compelling results from the
AEGIS trial, we look forward to the potential to provide the clinical
benefits of Soliris to significant numbers of Japanese patients with
PNH."
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. (2)
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. (2,7)
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 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 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.
Please see full prescribing information at www.soliris.net.
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
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, the risk that pending
litigation may be resolved adversely, 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.
(1) Socie G, Mary J Yves, de Gramont A, et al. Paroxysmal nocturnal
haemoglobinuria: long-term follow-up and prognostic factors. Lancet.
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(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.
SOURCE: Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Irving Adler, 203-272-8210
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