NICE Confirms AGNSS Positive Assessment that Eculizumab (Soliris®) is a Very Effective Treatment for aHUS Patients and Produces

  NICE Confirms AGNSS Positive Assessment that Eculizumab (Soliris®) is a Very
  Effective Treatment for aHUS Patients and Produces Substantial
  Quality-Adjusted Life Year Gains of a Magnitude Rarely Seen for a New Drug

  NICE Confirms up to 40% of aHUS Patients May Die or Progress to End-stage
Renal Failure and Require Dialysis with the First Clinical aHUS Manifestation
                              without Eculizumab

   NICE Confirms Current Broad NHS England Interim Commissioning Policy to
   Continue for Eculizumab Treatment of Existing and New Patients with aHUS

         NICE Seeks Further Information from NHS England and Alexion

      Alexion Remains Committed to Work Collaboratively with Government

Business Wire

CHESHIRE, Conn. -- March 3, 2014

Alexion Pharmaceuticals (Nasdaq:ALXN) is pleased that the National Institute
for Health and Clinical Excellence (NICE) Evaluation Committee has
acknowledged that eculizumab (Soliris^®) is a very effective treatment option
for patients with aHUS, and that the use of eculizumab would be of significant
value to patients with this life-threatening disorder who have no other
treatment options.

In an Evaluation Consultation Document (ECD) released today, the NICE
Evaluation Committee confirmed that currently available supportive care for
patients with aHUS has limited impact on disease morbidity and mortality, but
a substantial negative effect on a patient’s quality of life. Importantly, the
Committee underscored that substantial restoration of health for a very long
period is achieved with ongoing treatment with eculizumab. The Committee also
concluded that eculizumab produced a substantial gain in quality-adjusted life
years (QALY, a measure of the number of high-quality years of life gained) of
a magnitude rarely seen for a new drug treatment – let alone one for an
ultra-rare disorder.

Today’s very positive assessment by NICE of the clinical value of eculizumab
for patients with aHUS aligns with the two prior positive recommendations from
Government – AGNSS and NHS England. In addition, NICE confirmed today that the
broad interim NHS England commissioning policy for eculizumab treatment of
existing and new patients with aHUS will continue.

However, Alexion is concerned that nearly 3 years after Government commenced
its evaluation, and more than 1 year after eculizumab was referred to NICE,
the Committee was still unable to provide a formal recommendation whether
eculizumab should be nationally commissioned. Additionally, NICE has now
stated that it will seek further information from NHS England on what
considerations relating to the management of its specialised commissioning
budget it considers should be taken into account. NICE has also asked the
company to provide additional information related to budget impact. NICE has
not requested any additional clinical effectiveness information from Alexion.

Currently, new and existing patients with aHUS in England are able to receive
eculizumab through an interim policy commissioned by NHS England last year,
and NICE confirmed today that this interim policy will remain in place pending
the outcome of NICE’s appraisal. Alexion looks forward to confirmation by NICE
of a final policy so that patients with aHUS in England may continue to
receive treatment.

About Soliris

Soliris 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 indicated to inhibit complement-mediated TMA. The
effectiveness of Soliris in aHUS is based on its effects on TMA and renal
function. Prospective clinical trials in additional patients, the preliminary
results of which were reported at international nephrology and hematology
conferences in 2013, 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). For the
breakthrough innovation in complement inhibition, Alexion and Soliris have
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 U.S. prescribing information on Soliris is
available at

The full prescribing information on Soliris in Europe is available at:

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

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:


Safe Harbor Statement

This news release contains forward-looking statements, including statements
related to potential health and medical benefits of Soliris® (eculizumab) for
the treatment of patients with aHUS, pricing for Soliris in England, whether
eculizumab will be nationally commissioned in England and the timing of such
commissioning, and the continuation of existing programs in England that
provide access to Soliris. 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
reimbursement of Soliris, 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.


Alexion Pharmaceuticals
Irving Adler, 203-271-8210
Exec. Director, Corporate Communications
Kim Diamond, 917-699-5093
Senior Director, Corporate Communications
Rx Communications
Rhonda Chiger, 917-322-2569
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