FDA Grants Priority Review for Alexion's sBLA for Soliris® (eculizumab) as a Treatment for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)
CHESHIRE, Conn.--(BUSINESS WIRE)--
Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) announced today that the
U.S. Food and Drug Administration (FDA) has granted the Company's
request for Priority Review of its supplemental Biologics License
Application (sBLA) for Soliris® (eculizumab) as a treatment for patients
with atypical Hemolytic Uremic Syndrome (aHUS). A Priority Review
designation is given to drugs that may offer major advances in
treatment, or provide a treatment where no adequate therapy exists.
If approval is granted, Alexion anticipates that Soliris would be
available for U.S. patients with aHUS in the fourth quarter of 2011. The
European Medicines Agency (EMA) is also reviewing a marketing
application for Soliris as a treatment for patients with aHUS in the
Both the US and EU sBLA submissions include the positive data from the
two 26-week Phase 2 studies of Soliris as a treatment for adult and
adolescent patients with aHUS. Primary endpoints in both studies were
achieved with statistical significance. Final data from these studies
will be presented at the European Hematology Association (EHA) Congress,
to be held in London on June 9-12, 2011.1
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.2-4 Approximately 60 percent of patients with aHUS
require dialysis or a kidney transplant or die within a year of
diagnosis.5 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
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.7,8 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.
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, an
ultra-rare, debilitating 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
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.
Safe Harbor Statement
This news release contains forward-looking statements, including
statements related to anticipated clinical development, regulatory and
commercial 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-Q for
the period ended March 31, 2011, 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) Alexion Pharmaceuticals Corporate Website. Press release available
from URL: http://www.alxn.com/News/article.aspx?relid=580263
(2) 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
(3) Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin
Nephrol Hypertens 2010 May;19(3):242-7.
(4) Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney
Int 2006 Jul;70(1):16-23.
(5) Loirat C, Noris M, Fremeaux-Bacchi V. Complement and the atypical
hemolytic uremic syndrome in children. Pediatr Nephrol.
(6) 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.
(7) 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.
(8) 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.
Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Director, Corporate Communications and Public Policy
Mark Marmur, 609-354-8135
Rhonda Chiger, 917-322-2569
Source: Alexion Pharmaceuticals, Inc.
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