Alexion's Soliris® (eculizumab) Receives Marketing Approval in Japan for All Patients with aHUS

  Alexion's Soliris® (eculizumab) Receives Marketing Approval in Japan for All
  Patients with aHUS

First and Only Approved Treatment in Japan for Patients Suffering from aHUS, a
               Chronic and Life-Threatening Ultra-Rare Disease

Business Wire

CHESHIRE, Conn. -- September 13, 2013

Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN), today announced that the Ministry
of Health, Labour and Welfare (MHLW) in Japan has approved the use of Soliris®
(eculizumab) for the treatment of pediatric and adult patients with atypical
hemolytic uremic syndrome (aHUS), a life-threatening ultra-rare disorder.
Soliris is already approved in Japan for paroxysmal nocturnal hemoglobinuria
(PNH), another severe and ultra-rare disease. Alexion expects that initial
patients with aHUS in Japan will commence treatment with Soliris during the
fourth quarter of this year.

aHUS is a life-threatening and ultra-rare chronic genetic disease that can
progressively damage vital organs, leading to stroke, heart attack, kidney
failure, and death.^1 The morbidities and premature mortality in aHUS is
caused by chronic uncontrolled activation of the complement system, resulting
in thromobotic microangiopathy (TMA, the formation of blood clots in small
blood vessels throughout the body).^2,3 Soliris, a first-in-class terminal
complement inhibitor, specifically targets uncontrolled complement activation,
and is approved for the suppression of TMA in patients with aHUS.

"Soliris has been proven to have a life-transforming impact on patients
suffering from aHUS and represents a significant step forward in the treatment
of this devastating disease," said Prof. Motoshi Hattori, Professor and
Director of the Department of Pediatric Nephrology, Tokyo Women’s Medical
University of Tokyo Woman’s UHP. "Results from clinical studies show that
chronic Soliris treatment inhibits complement-mediated TMA which is
responsible for thrombosis, vital organ failure, and other life-threatening
manifestations of aHUS.”

"The approval of Soliris for the treatment of aHUS by the Japanese government
brings life-transforming hope to patients suffering from this devastating
disease," said David Hallal, Executive Vice President and Chief Commercial
Officer of Alexion. "We will initiate our disease awareness and education
programs as we work closely with the healthcare community to support rapid and
accurate diagnosis of aHUS as well as better informed treatment decisions."

Soliris is also approved by the U.S. Food and Drug Administration (FDA), the
European Medicines Agency (EMA) and other regulatory authorities for the
treatment of patients with aHUS to inhibit complement-mediated TMA. In
addition, Soliris is approved in more than 40 countries, including Japan, for
the treatment of patients with PNH, a debilitating, ultra-rare and
life-threatening blood disorder.

Clinical Data

Alexion’s supplemental new drug application (sNDA) for Soliris in Japan
included clinical data from two prospective, controlled, open-label studies in
adolescent and adult patients with aHUS, and a third retrospective study in
pediatric patients with aHUS, which together included a broad range of aHUS
patients in North America and Europe. In these studies, all patients treated
with Soliris demonstrated rapid and sustained reduction in terminal complement
activity, and chronic administration of Soliris resulted in rapid and
sustained reduction in complement-mediated TMA. Soliris was well tolerated in
these clinical studies. The most frequently reported adverse events were
hypertension, upper respiratory tract infection, and diarrhea. In addition,
the sNDA in Japan included data from a retrospective study and from an ongoing
prospective study of both pediatric and adult aHUS patients in Japan. Results
from these studies demonstrated that the safety and efficacy of Soliris in
these Japanese patients with aHUS were consistent with those noted in the
clinical studies of Soliris in aHUS patients in North America and Europe.

About aHUS

aHUS is a chronic, ultra-rare, and life-threatening disease in which a genetic
deficiency in one or more complement regulatory genes causes chronic
uncontrolled complement activation, resulting in complement-mediated
thrombotic microangiopathy (TMA), the formation of blood clots in small blood
vessels throughout the body.^4,5 Permanent, uncontrolled complement activation
in aHUS causes a life-long risk for TMA, which leads to sudden, catastrophic,
and life-threatening damage to the kidney, brain, heart, and other vital
organs, and premature death.^4,6 [ ]Sixty-five percent of all patients with
aHUS die, require kidney dialysis, or have permanent kidney damage within the
first year after diagnosis despite plasma exchange or plasma infusion
(PE/PI).^3,7 The majority of patients with aHUS who receive a kidney
transplant commonly experience subsequent systemic TMA, resulting in a 90%
transplant failure rate in these TMA patients.^8

aHUS affects both children and adults. Complement-mediated TMA also causes
reduction in platelet count (thrombocytopenia) and red blood cell destruction
(hemolysis). While mutations have been identified in at least ten different
complement regulatory genes, mutations are not identified in 30-50% of
patients with a confirmed diagnosis of aHUS.^2

About Soliris

Soliris is a first-in-class terminal complement inhibitor developed from the
laboratory through regulatory approval and commercialization by Alexion.
Soliris is also approved in the US, European Union, Japan and other countries
as the first and only treatment for patients with paroxysmal nocturnal
hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood
disorder, characterized by complement-mediated hemolysis (destruction of red
blood cells).

Soliris is also approved in the US, the European Union, Japan and other
territories as the first and only treatment for patients with atypical
hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic
microangiopathy, a debilitating, ultra-rare and life-threatening genetic
disorder characterized by complement-mediated thrombotic microangiopathy
(blood clots in small vessels). The effectiveness of Soliris in aHUS is based
on the effects on thrombotic microangiopathy (TMA) and renal function.
Prospective clinical trials in additional patients are ongoing to confirm the
benefit of Soliris in patients with aHUS. Soliris is not indicated for the
treatment of patients with Shiga toxin E. coli related hemolytic uremic
syndrome (STEC-HUS).

Alexion's breakthrough approach in complement inhibition has received 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.

Important Safety Information

The US product label for Soliris includes a boxed warning: "Life-threatening
and fatal meningococcal infections have occurred in patients treated with
Soliris. Meningococcal infection may become rapidly life-threatening or fatal
if not recognized and treated early. Comply with the most current Advisory
Committee on Immunization Practices (ACIP) recommendations for meningococcal
vaccination in patients with complement deficiencies. Immunize patients with a
meningococcal vaccine at least 2 weeks prior to administering the first dose
of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of
developing a meningococcal infection. (See Serious Meningococcal Infections
(5.1) for additional guidance on the management of meningococcal infection.)
Monitor patients for early signs of meningococcal infections and evaluate
immediately if infection is suspected. Soliris is available only through a
restricted program under a Risk Evaluation and Mitigation Strategy (REMS).
Under the Soliris REMS, prescribers must enroll in the program. Enrollment in
the Soliris REMS program and additional information are available by
telephone: 1-888-soliris (1-888-765-4747)."

In patients with PNH, the most frequently reported adverse events observed
with Soliris treatment in clinical studies were headache, nasopharyngitis
(runny nose), back pain and nausea. Soliris treatment of patients with PNH
should not alter anticoagulant management because the effect of withdrawal of
anticoagulant therapy during Soliris treatment has not been established. In
patients with aHUS, the most frequently reported adverse events observed with
Soliris treatment in clinical studies were hypertension, upper respiratory
tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract
infection, and leukopenia. Please see full prescribing information for
Soliris, including boxed WARNING regarding risk of serious meningococcal

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 and aHUS,
two debilitating, ultra-rare and life-threatening disorders caused by chronic
uncontrolled complement activation. Soliris is currently approved in more than
40 countries for the treatment of PNH, and in the United States, Europe, Japan
and other territories for the treatment of aHUS. Alexion is evaluating other
potential indications for Soliris and is pursuing development of four other
innovative biotechnology product candidates which are being investigated
across additional severe and ultra-rare disorders beyond PNH and aHUS. This
press release and further information about Alexion Pharmaceuticals, Inc. can
be found at


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-Q for
the period ended June 30, 2013, 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) Noris M, Remuzzi G. Atypical hemolytic-uremic syndrome. N Engl J Med.

(2) Noris M, Caprioli J, Bresin E, et al. Relative role of genetic complement
abnormalities in sporadic and familial aHUS and their impact on clinical
phenotype. Clin J Am Soc Nephrol. 2010;5:1844-59.

(3) Caprioli J, Noris M, Brioschi S, et al. The impact of MCP, CFH, and IF
mutations on clinical presentation, response to treatment, and outcome. Blood.

(4) Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin
Nephrol Hypertens. 2010;19(3):242-7.

(5) Ariceta G, Besbas N, Johnson S, et al. Guideline for the investigation and
initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr
Nephrol. 2009;24:687-96.

(6) Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int.

(7) Loirat C, Garnier A, Sellier-Leclerc AL, Kwon T. Plasmatherapy in atypical
hemolytic uremic syndrome. Semin Thromb Hemost. 2010;36:673-81.

(8) Bresin E, Daina E, Noris M, 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;1:88-99.


Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Executive Director, Corporate Communications
Alexion Pharmaceuticals, Inc.
Kim Diamond, 203-439-9600
Senior Director, Corporate Communications
Rx Communications
Rhonda Chiger, 917-322-2569
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