Alexion Submits Applications for Soliris® (Eculizumab) as a Treatment for Patients with Atypical Hemolytic Uremic Syndrome (aHUS) in the United States and European Union
2011 Revenue Guidance Revised Upward
CHESHIRE, Conn.--(BUSINESS WIRE)--
Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) announced today that the
company has submitted marketing applications to the U.S. Food and Drug
Administration (FDA) and the European Medicines Agency (EMA) for Soliris®
(eculizumab) as a treatment for patients with atypical Hemolytic Uremic
Syndrome (aHUS).
Both the US and EU filings include the positive data from the two
26-week Phase 2 studies of Soliris as a treatment for adult and
adolescent patients with aHUS. Preliminary data from these two studies
were presented at the American Society of Nephrology (ASN) annual
meeting in November 2010. 1-3 Primary endpoints in both
studies were achieved with statistical significance. aHUS is an
ultra-rare, chronic and life-threatening disease in which uncontrolled
complement activation causes blood clots in small blood vessels
throughout the body (thrombotic microangiopathy, or TMA) leading to
kidney failure, stroke, heart attack and death.
"The US and EU regulatory submissions put us one step closer toward
accomplishing our goal to transform the lives of patients suffering with
aHUS," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "We
recognize that patients with aHUS, a life-threatening and ultra-rare
disorder, lack adequate treatment options, and we look forward to
working with regulatory authorities as they review our applications."
Updated 2011 Revenue Guidance
Alexion is updating its 2011 revenue guidance, from the previously
announced range of $715- $735 million, now to the higher range of $720
to $740 million. Other items of previously announced 2011 guidance are
being reiterated at this time.
The upward revision in revenue guidance takes into account continued
global growth of Soliris for PNH, and the potential for an earlier than
previously expected US launch of Soliris for aHUS. The earlier launch
could occur if the US regulatory submission is accepted for priority
review by the FDA and if a positive decision is then received, making a
launch possible late in the fourth quarter of 2011. Further, the upward
revision takes into account the potential for disruptions in treatment
for individual patients in Japan associated with the recent natural
disasters in that country.
About aHUS
aHUS is a chronic, ultra-rare disease characterized by thrombotic
microangiopathy (TMA), the formation of blood clots in small blood
vessels throughout the body, causing a reduction in platelet count and
life-threatening damage to the kidney, brain, heart and other vital
organs.4-6 Approximately 60 percent of patients with aHUS
require dialysis or a kidney transplant or die within a year of
diagnosis.7 The majority of patients with aHUS who receive a
kidney transplant experience severe complications of the disease, and
more than 90 percent of these patients experience failure of the donor
kidney.8
aHUS is a progressive disease caused by life-long uncontrolled
activation of the complement system due to deficiencies in complement
regulatory genes. With genetic deficiency of naturally occurring
complement inhibitors, patients experience chronic uncontrolled
activation of the complement system, causing ongoing inflammation and
blood clots in vital organs.9,10 In patients with aHUS,
uncontrolled complement activation results in an ongoing risk of sudden
and catastrophic life-threatening complications. Currently, mutations
have been identified in at least ten different genes; however, in
approximately one-half of patients diagnosed with aHUS, the specific
genetic deficiency cannot currently be identified.
About Soliris
Soliris is a first-in-class terminal complement inhibitor developed from
the laboratory through regulatory approval and commercialization by
Alexion. Soliris has been approved in the U.S., European Union, Japan
and other territories as the first treatment for patients with PNH, a
debilitating, ultra-rare and life-threatening blood disorder defined by
chronic uncontrolled complement activation which causes chronic red
blood cell destruction (hemolysis), leading to blood clots, organ
failure, and shortened survival. Prior to these approvals, there were no
therapies specifically available for the treatment of patients with PNH.
Soliris (eculizumab) is not approved for the treatment of aHUS or other
indications other than PNH. Alexion's breakthrough 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 in patients with PNH. The most
frequent adverse events observed in clinical studies of patients with
PNH were headache, nasopharyngitis (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 PNH 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 focused on
serving patients with severe and ultra-rare disorders through the
innovation, development and commercialization of life-transforming
therapeutic products. Alexion is the global leader in complement
inhibition, and has developed and markets Soliris®
(eculizumab) as a treatment for patients with PNH, a debilitating,
ultra-rare and life-threatening blood disorder. Soliris is approved in
more than 35 countries. Alexion is evaluating other potential
indications for Soliris and is pursuing development of other innovative
biotechnology 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 anticipated clinical development milestones and
potential health and medical benefits of Soliris®
(eculizumab) for the potential treatment of patients with aHUS. 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 for its current or potential new
indications, 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, 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.
References
(1) Abstract 1338 entitled "Safety and Efficacy of Eculizumab in aHUS
Patients Resistant to Plasma Therapy: Interim Analysis from a Phase II
Trial," presented in an oral presentation at the American Society of
Nephrology (ASN) Annual Meeting on November 20, 2010 by Dr. Christophe
Legendre.
(2) Alexion Pharmaceuticals Corporate Website. Press release available
from URL: http://www.alxn.com/News/article.aspx?relid=532082.
(3) Abstract 157 entitled "Safety and Efficacy of Eculizumab in aHUS
Patients on Chronic Plasma Therapy: Interim Analysis of a Phase II
Trial," presented in a poster presentation at the American Society of
Nephrology (ASN) Annual Meeting on November 19, 2010 by Dr. Petra Muus.
(4) Alexion Pharmaceuticals. Soliris in Other Kidney Disorders. Alexion
Pharmaceuticals Corporate Website 2010 September 24 Available from: URL: http://www.alexionpharma.com/RandD/Soliris%20In%20Other/kidney%20disorders.aspx#A1.
(5) Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin
Nephrol Hypertens 2010 May;19(3):242-7.
(6) Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney
Int 2006 Jul;70(1):16-23.
(7) Loirat C, Noris M, Fremeaux-Bacchi V. Complement and the atypical
hemolytic uremic syndrome in children. Pediatr Nephrol.
2008;23(11):1957-1972.
(8) Bresin E, Daina E, Noris M, Castelletti F, Stefanov R, Hill P, et
al. Outcome of renal transplantation in patients with non-Shiga
toxin-associated hemolytic uremic syndrome: prognostic significance of
genetic background. Clin J Am Soc Nephrol 2006 Jan;1(1):88-99.
(9) Hosler GA, Cusumano AM, Hutchins GM. Thrombotic thrombocytopenic
purpura and hemolytic uremic syndrome are distinct pathologic entities.
A review of 56 autopsy cases. Arch Pathol Lab Med 2003 Jul;127(7):834-9.
(10) Ståhl A, Vaziri-Sani F, Heinen S, Kristoffersson A-C, Gydell K-H,
Raafat R, Gutierrez A, Beringer O, Zipfel PF, and Karpman D. Factor H
dysfunction in patients with atypical hemolytic uremic syndrome
contributes to complement deposition on platelets and their activation.
Blood. 2008;111:5307-5315.
Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Senior
Director, Corporate Communications and Public Policy
or
Media:
Makovsky
& Company
Mark Marmur, 609-354-8135
or
Investors:
Rx
Communications
Rhonda Chiger, 917-322-2569
Source: Alexion Pharmaceuticals, Inc.
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