Alexion's Soliris® (eculizumab) Receives Marketing Approval in Australia for All Patients with PNH
CHESHIRE, Conn., Feb 25, 2009 (BUSINESS WIRE) -- Alexion Pharmaceuticals, Inc., (Nasdaq:ALXN) today announced that the
Australian Government's Therapeutic Goods Administration has approved
the use of Soliris(R) (eculizumab) for the treatment of all patients in
Australia 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 Australia for the treatment of PNH.
"Prior to this positive action by the government, there has not been an
approved, safe and effective therapy for the treatment of patients with
PNH in Australia," said Professor Jeffrey Szer, M.B., B.S., FRACP,
Director of Clinical Haematology, The Royal Melbourne Hospital. "Soliris
reduced hemolysis in every patient treated in clinical studies.
Regulatory approval is an important step towards providing Australian
patients with this debilitating and life-threatening disease access to
Soliris, the only approved treatment. With access to Soliris, physicians
will be able to prevent the hemolysis that underlies the severe,
progressive and life-threatening consequences of PNH."
The marketing application submitted to the Australian regulatory
authorities 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, and by Health Canada
in 2009, using data from the same studies. Soliris is currently being
used to treat patients with PNH in more than 18 countries.
"This marketing approval reflects Australia's recognition of the safety
and efficacy of Soliris as a treatment for patients with PNH," said
Leonard Bell, M.D., Chief Executive Officer of Alexion. "Our
Sydney-based organization will now work with the Australian Federal
healthcare authorities to obtain reimbursement for Soliris so that
Australian patients with PNH who can benefit from Soliris will have
access to it." A decision regarding funding of Soliris therapy for
eligible patients is expected by the end of 2009.
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)
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 Australian 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. The Registry
will be an integral part of Alexion's ongoing commitment to the
Australian authorities and will enable monitoring and data collection
regarding the safety and effectiveness of Soliris.
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
Australia. 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) Hillmen P, Young NS, Schubert J, et al. The complement inhibitor
eculizumab in paroxysmal nocturnal hemoglobinuria. NEngl J Med.
2006;355:1233-1243.
(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) Socie G, Hillmen P, Muus P et al. Sustained improvements in
transfusion requirements, fatigue and thrombosis with eculizumab
treatment in paroxysmal nocturnal hemoglobinuria [abstract]. Blood.
2007; 110: A3672.
(4) Socie 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. NEngl 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, Sr. Director Corporate Communications
203-271-8210
Makovsky & Company
Mark Marmur (Media)
212-508-9670
Rx Communications
Rhonda Chiger (Investors)
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