Alexion Joins the International Federation of Kidney Foundations in Celebrating World Kidney Day 2013

  Alexion Joins the International Federation of Kidney Foundations in
  Celebrating World Kidney Day 2013

   -- Global Initiative to Raise Awareness of the Importance of Kidneys to
          Overall Health and Reduce the Impact of Kidney Diseases --

Business Wire

CHESHIRE, Conn. -- March 14, 2013

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced its support of
World Kidney Day 2013, a global awareness campaign led by the International
Society of Nephrology (ISN) and the International Federation of Kidney
Foundations. Celebrated on the second Thursday in March each year, the mission
of World Kidney Day is to raise awareness of the importance of our kidneys to
overall health and to reduce the incidence and impact of kidney disease and
its associated health problems worldwide. This year’s campaign -“Kidney’s for
Life - Stop Kidney Attack!” - aims to educate the general public and the
healthcare community about diseases that affect the kidneys and the importance
of early detection and intervention. Alexion is pleased to sponsor this
initiative and joins other leading companies engaged in researching and
delivering innovative therapies to improve the lives of patients suffering
from life-threatening disorders, including those that impact the kidneys.

Many health conditions that damage kidney function can severely affect a
patient’s quality of life and progress to end-stage renal disease (ESRD) and
premature death. One such disease is atypical hemolytic uremic syndrome
(aHUS), a chronic, life-threatening ultra-rare disease that can progressively
damage vital organs, including the kidney. Patients with aHUS experience
kidney damage and kidney failure leading to ESRD.^1 Sixty-five percent of
patients with aHUS require kidney dialysis, have permanent kidney damage or
die within the first year after diagnosis despite plasma exchange or plasma
infusion (PE/PI).^2,3

"At the age of 28, I was experiencing renal failure but my doctors were not
able to pinpoint the cause of it,” says Jill Ziegler, a young adult diagnosed
with aHUS. “Subsequently, I was diagnosed with a severe and life-threatening
condition that is so rare that many nephrologists have never seen a case of
it. I tried to maintain some semblance of a normal life with my family, but I
remained in end stage kidney failure and on permanent dialysis. Fortunately, I
was able to have a kidney transplant and receive a new treatment for aHUS.
Today I want others with aHUS to know that help is out there and they are not

“We know first-hand the importance of the disease awareness efforts taking
part on World Kidney Day 2013. In serving patients with aHUS, we are
continually reminded of the devastation that kidney damage causes and the
importance of disease education to facilitate early diagnosis and timely
intervention,” said Stephen P. Squinto, PhD, Executive Vice President and Head
of Research and Development of Alexion. "We are pleased to support the
International Society of Nephrology and the International Federation of Kidney
Foundations in their efforts to help patients with other severe diseases that
affect the kidneys.”

In addition to its work in aHUS, Alexion is conducting investigational studies
in kidney transplant patients who are at elevated risk for antibody mediated
rejection, or other kidney transplant patients who are at elevated risk for
delayed graft function.

For more information about World Kidney Day, visit

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
require kidney dialysis, have permanent kidney damage or die within the first
year after diagnosis despite plasma exchange or plasma infusion (PE/PI).^2,3
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.^7

aHUS affects both children and adults.^8 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.^8

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 a treatment for patients with PNH and aHUS, two debilitating,
ultra-rare and life-threatening disorders caused by chronic uncontrolled
complement activation. The treatment 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 its marketed drug and is developing four other highly
innovative biotechnology product candidates, which are being investigated
across nine severe and ultra-rare disorders beyond PNH and aHUS. This press
release and further information about Alexion Pharmaceuticals, Inc. can be
found at:



     Noris M, Bresin E, Mele C, et al. Atypical Hemolytic-Uremic Syndrome.
     2007 Nov 16 [Updated 2011 Mar 10]. In: Pagon RA, Bird TD, Dolan CR, et
1.  al., editors. GeneReviews™ [Internet]. Seattle (WA): University of
     Washington, Seattle; 1993-. Available from:
     Caprioli J, Noris M, Brioschi S, et al. The impact of MCP, CFH, and IF
2.   mutations on clinical presentation, response to treatment, and outcome.
     Blood. 2006;108:1267-1269.
3.   Loirat C, Garnier A, Sellier-Leclerc AL, Kwon T. Plasmatherapy in
     atypical hemolytic uremic syndrome. Semin Thromb Hemost. 2010;36:673-81.
     Definition from the UK National Institute for Clinical Effectiveness
     (NICE). 2004. Citizen Council Report on Ultra-Orphan Drugs. Available at and as defined in the following documents:
4.   Wales Medicines Strategy Group (AWMSG); Recommendations for a Belgian
     Plan for Rare Diseases; the EMINET Report commissioned by the European
     Commission’s Directorate General Enterprise and Industry, the European
     Union Committee of Experts on Rare Diseases’ (EUCERD)
5.   Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin
     Nephrol Hypertens. 2010;19(3):242-7.
     Ariceta G, Besbas N, Johnson S, et al. Guideline for the investigation
6.   and initial therapy of diarrhea-negative hemolytic uremic syndrome.
     Pediatr Nephrol. 2009;24:687-96.
7.   Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int.
     Bresin E, Daina E, Noris M, et al. Outcome of renal transplantation in
8.   patients with non-Shiga toxin-associated hemolytic uremic syndrome:
     prognostic significance of genetic background. Clin J Am Soc Nephrol.


Alexion Pharmaceuticals, Inc.:
Irving Adler, 203-272-259
Executive Director, Corporate Communications
Kim Diamond, 203-439-9600 (media)
Director, Corporate Communications
Press spacebar to pause and continue. Press esc to stop.