AGNSS Recommends Soliris® (eculizumab) for aHUS and Concludes “Eculizumab would help save lives and improve the quality of

  AGNSS Recommends Soliris® (eculizumab) for aHUS and Concludes “Eculizumab
  would help save lives and improve the quality of life for children and
  adults with atypical haemolytic uraemic syndrome”

  Health Ministers Decide to Not Follow AGNSS Recommendation for Soliris for
                                     aHUS

     Alexion Calls Upon Ministers to Reverse the Decision and Accept the
  Recommendation of AGNSS in the Best Interests of aHUS Patients in England

Business Wire

WEYBRIDGE, England -- January 23, 2013

Alexion Pharma UK, a subsidiary of Alexion Pharmaceuticals, Inc., has been
informed by the National Specialised Commissioning Team (NSCT) that the
Ministers of Health have decided to not follow a positive recommendation by
the Advisory Group for National Specialised Services (AGNSS) when assessing
Soliris® (eculizumab) as a treatment for patients with atypical haemolytic
uraemic syndrome (aHUS). It is important to underscore that the UK Government
developed a specific process and authorised a committee, AGNSS, to evaluate
treatments addressing patients with very rare disorders, and that AGNSS
concluded that “eculizumab should be routinely nationally commissioned for
patients with aHUS.” AGNSS further stated that, “Eculizumab would help save
lives and improve the quality of life for children (among whom the condition
is particularly prevalent) and adults with atypical haemolytic uraemic
syndrome.” However, the Ministers of Health have overruled this expert
committee recommendation and instead referred consideration of Soliris for the
treatment of patients with aHUS to the National Institute for Health and
Clinical Excellence (NICE).

aHUS is an unpredictable and life-threatening disease that affects both
children and adults. More than 50% of patients with aHUS experience
devastating consequences, including kidney failure, require dialysis or die
within 1 year of diagnosis.^1,2 Soliris was approved by the European
Commission in 2011 specifically for the treatment of patients suffering with
aHUS and is the only approved treatment option available. The Health Ministers
decision to refer the Soliris assessment to NICE comes over one and one-half
years after the initial application was submitted by the lead physician expert
at Newcastle-upon Tyne Hospitals NHS Foundation Trust, and following the
positive recommendation by the authorised committee AGNSS. This action pushes
the Health Ministers decision further into the future, and with an indefinite
timeframe, despite abundant clinical evidence on the safety and efficacy of
Soliris for these patients. Alexion is gravely disappointed by the
announcement and the underlying departure by the UK Government from an agreed
formal process. The significant delay in the Health Ministers decision
directly threatens the lives of aHUS patients in England by causing further
and unnecessary delays in access to safe and effective treatment.

Alexion is committed to working with the Health Ministers to make Soliris
available to children and adults as quickly as possible, and calls upon the
Ministers to accept the recommendation of the significant number of experts
and lay people called upon to review Soliris without delay, and to reverse his
decision and accept the recommendation of AGNSS in the best interests of aHUS
patients in England.

Background

It is important to underscore that the Government developed a specific process
and authorised the committee, AGNSS, to evaluate treatments addressing
patients with very rare disorders, and that AGNSS concluded that “eculizumab
should be routinely nationally commissioned for patients with aHUS.” AGNSS
further stated that, “Eculizumab would help save lives and improve the quality
of life for children (among whom the condition is particularly prevalent) and
adults with atypical haemolytic uraemic syndrome.” Specifically, in June 2011,
Newcastle-upon Tyne Hospitals NHS Foundation Trust submitted an application
for national commissioning of a service for patients with aHUS, and the
application was accepted by AGNSS. In January 2012, Alexion submitted a full
dossier of data and a complete assessment to AGNSS as part of a well-defined
process for review. The dossier included strong clinical data from the aHUS
registration trials, demonstrating that 100% of patients had a response to
therapy and that Soliris had a significant beneficial impact on TMA and
patient morbidities for patients with aHUS. Based on these data, and as
communicated on January 22 by the NCST to Alexion, AGNSS recommended to the
Health Minister that “eculizumab should be routinely nationally commissioned
for patients with aHUS”; yet the Minister chose instead to defer the decision
to a new process that is neither yet defined nor will apparently commence
until April 2013 at the earliest. In the meantime, children and adults in
England suffering from a life-threatening and ultra-rare condition continue to
wait while other patients with aHUS around the world are receiving treatment
and hope for a better life.

aHUS is an ultra-rare, life-threatening, chronic, genetic disease that
progressively damages vital organs, often leading to sudden and catastrophic
damage to the kidney, brain, heart, and other vital organs, and premature
death.^3-5 More than 50% of patients have kidney failure, require dialysis or
die within 1 year of diagnosis.^1,2 In November 2011, the European Commission
approved Soliris as the first and only treatment for children and adults with
aHUS.

References

1. Caprioli J, Noris M, Brioschi S, et al for the International Registry of
Recurrent and Familial HUS/TTP (2006). Genetics of HUS: the impact of MCP,
CFH, and IF mutations on clinical presentation, response to treatment, and
outcome. Blood.108:1267-1279

2. Loirat C, Garnier A, Sellier-Leclerc AL et el (2010). Plasmatherpay in
atypical hemolytic uremic syndrome., Semin Thromb Hemost. 2010;36:673-681

3. Noris M, Remuzzi G (2009). Atypical hemolytic-uremic syndrome. N Engl J Med
361:1676-87

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

5. Tsai HM (2006). The molecular biology of thrombotic microangiopathy. Kidney
Int ;70(1):16-23

Notes to Editors:

About Soliris

Soliris is a first-in-class terminal complement inhibitor developed from the
laboratory through regulatory approval and commercialisation by Alexion.
Soliris is 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, characterised by complement-mediated hemolysis (destruction of red
blood cells). In PNH evidence of clinical benefit of Soliris is limited to
patients with a history of transfusions. Soliris is also approved in the US 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 characterised
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. Alexion's breakthrough approach in complement inhibition
has received the pharmaceutical industry's highest honours: 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:

http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/000791/human_med_001055.jsp&mid=WC0b01ac058001d124.

Important Safety Information

In Europe, the Summary of Product Characteristics (SmPC) for Soliris includes
a special warning and precaution for use: "Due to its mechanism of action, the
use of Soliris increases the patient's susceptibility to meningococcal
infection (Neisseria meningitidis). These patients might be at risk of disease
by uncommon serogroups (particularly Y, W135 and X), although meningococcal
disease due to any serogroup may occur. To reduce the risk of infection, all
patients must be vaccinated at least 2 weeks prior to receiving Soliris. PNH
patients must be vaccinated 2 weeks prior to Soliris initiation. aHUS patients
who are treated with Soliris less than 2 weeks after receiving a meningococcal
vaccine must receive treatment with appropriate prophylactic antibiotics until
2 weeks after vaccination. Patients must be re-vaccinated according to current
medical guidelines for vaccination use. Tetravalent vaccines against serotypes
A, C, Y and W135 are strongly recommended, preferably conjugated ones.
Vaccination may not be sufficient to prevent meningococcal infection.
Consideration should be given to official guidance on the appropriate use of
antibacterial agents. Cases of serious or fatal meningococcal infections have
been reported in Soliris treated patients. All patients should be monitored
for early signs of meningococcal infection, evaluated immediately if infection
is suspected, and treated with antibiotics if necessary. Patients should be
informed of these signs and symptoms and steps taken to seek medical care
immediately." Physicians must discuss the benefits and risks of Soliris
therapy with patients and provide them with a patient information brochure and
a patient safety card.

The most common or serious adverse reactions are headache (occurred mostly in
the initial phase), leukopenia and meningococcal infection. The most common
(>10%) adverse reactions reported in paediatric aHUS patients were diarrhoea,
vomiting, pyrexia, upper respiratory tract infection and headache. Soliris
treatment should not alter anticoagulant management. Please see Summary of
Product Characteristics for full prescribing information for Soliris,
including information regarding risk of serious meningococcal infection.

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on
serving patients with severe and ultra-rare disorders through the innovation,
development and commercialisation 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 and the
European Union for the treatment of aHUS. Alexion is evaluating other
potential indications for Soliris and is developing four other highly
innovative biotechnology product candidates.

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
September 30, 2012, 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.

Contact:

For further information please contact
Red Door Communications
Laura Good, +44 (0) 20 8392 8054 / +44 (0) 7889 757 191
lgood@rdcomms.com
or
Alexion Pharmaceuticals, Inc. (Media)
Kim Diamond, +00 (1) 203 439 9600
Director, Corporate Communications
or
Irving Adler, +00 (1) 914-584-4948
Executive Director of Corporate Communications