European Commission Grants Orphan Drug Designation to Soliris® (Eculizumab) for the Prevention of Graft Rejection Following

  European Commission Grants Orphan Drug Designation to Soliris® (Eculizumab)
  for the Prevention of Graft Rejection Following Solid Organ Transplantation

Business Wire

CHESHIRE, Conn. -- April 23, 2014

Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) today announced that the European
Commission has granted an orphan drug designation (ODD) to Soliris^®
(eculizumab), a first-in-class terminal complement inhibitor, for the
prevention of graft rejection following solid organ transplantation. Graft
rejection can cause severe injury to the transplanted organ and is a
significant barrier to successful transplantation.

Soliris is currently approved in the United States, European Union, Japan and
other countries for the treatment of patients with paroxysmal nocturnal
hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two
debilitating, ultra-rare and life-threatening disorders caused by chronic
uncontrolled complement activation. Alexion is investigating Soliris for the
prevention of acute antibody-mediated rejection (AMR) in kidney transplant
recipients, and for the prevention of delayed graft function (DGF) in patients
receiving deceased donor kidney transplants. Soliris is not approved in any
country to prevent or treat rejection following kidney or other solid organ
transplantation.

“Rejection after transplantation is a severe and potentially devastating
occurrence for patients undergoing organ transplantation due to the very real
risk of losing the transplanted organ,” said Martin Mackay, Ph.D., Executive
Vice President, Global Head of R&D at Alexion. “By specifically inhibiting the
terminal complement pathway, Soliris has the potential to lower the risk of
rejection, a benefit that could lead to improved clinical outcomes for these
patients.”

The European Commission grants orphan medicinal product status to provide
incentives to develop medicinal products to treat, prevent or diagnose
diseases or conditions that affect no more than five in 10,000 persons in the
EU. The orphan medicinal product status designation would provide Alexion with
certain benefits and incentives, including a period of marketing exclusivity
if Soliris is approved in the EU for the orphan therapeutic indication of
acute antibody-mediated rejection (AMR).

About AMR

Acute antibody-mediated rejection (AMR) is a severe and potentially
life-threatening condition that can lead to severe allograft damage resulting
in rapid loss of function and possible loss of the transplanted organ.^1
Patients who are sensitized (have high levels of donor-specific-antibodies
[DSAs]) are at high risk for developing acute AMR,^1,2 may have difficulty
finding a donor to who they are not sensitized, and therefore may never have
the opportunity to have a life-saving transplant. The development of acute AMR
is believed to be primarily a result of uncontrolled complement activation
caused by DSAs.^1,2 Currently, there are no approved therapies for the
prevention of acute AMR.

About Soliris

Soliris^® (eculizumab) is a first-in-class terminal complement inhibitor
developed from the laboratory through regulatory approval and
commercialization by Alexion. Soliris is approved in the U.S. (2007), European
Union (2007), Japan (2010) 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
indicated to reduce hemolysis. Soliris is also approved in the U.S. (2011),
the European Union (2011), Japan (2013) and other countries as the first and
only treatment for patients with atypical hemolytic uremic syndrome (aHUS), a
debilitating, ultra-rare and life-threatening genetic disorder characterized
by complement-mediated thrombotic microangiopathy, or TMA (blood clots in
small vessels). Soliris is not approved to prevent or treat rejection
following kidney or other solid organ transplantation.

Alexion's breakthrough approach in terminal 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. More information, including the full prescribing information on
Soliris, is available at www.soliris.net.

Important Safety Information

The U.S. 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 two 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
infection.

About Alexion

Alexion is a biopharmaceutical company focused on serving patients with severe
and 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 nearly 50 countries for the
treatment of PNH, and in the United States, European Union, Japan and other
countries for the treatment of aHUS. Alexion is evaluating other potential
indications for Soliris in additional severe and ultra-rare disorders beyond
PNH and aHUS, and is developing other highly innovative biotechnology product
candidates across multiple therapeutic areas. This press release and further
information about Alexion can be found at: www.alexionpharma.com.

[ALXN-G]

Safe Harbor Statement

This news release contains forward-looking statements, including statements
related to potential medical benefits of Soliris^® (eculizumab) for the
prevention of rejection after solid organ transplant. 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 rejection after solid organ transplant and the timing
of such decisions, delays in arranging satisfactory manufacturing capabilities
and establishing commercial infrastructure for Soliris for rejection after
solid organ transplant, the possibility that results of clinical trials are
not predictive of safety and efficacy results of Soliris for rejection after
solid organ transplant, the risk that third party payors (including
governmental agencies) will not reimburse for the use of Soliris for rejection
after solid organ transplant (if approved) at acceptable rates or at all, the
risk that estimates regarding the number of patients with Soliris for
rejection after solid organ transplant and observations regarding the natural
history of patients with Soliris for rejection after solid organ transplant
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 Annual Report on Form 10-K for
the period ended Dec. 31, 2013. 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. Takemoto SK, Zeevi A, Feng S, et al. National conference to assess
antibody-mediated rejection in solid organ transplantation. Am J Transplant.
2004; 4(7):1033-41.

2. Collins AB, Schneeberger EE, Pascual MA, et al. Complement activation in
acute humoral renal allograft rejection: diagnostic significance of C4d
deposits in peritubular capillaries. J Am Soc Nephrol. 1999;10(10):2208-14.

Contact:

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