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

Thu Mar 14, 2013 6:30am EDT

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-- Global Initiative to Raise Awareness of the Importance of Kidneys to Overall
Health and Reduce the Impact of Kidney Diseases --
CHESHIRE, Conn.--(Business Wire)--
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 alone." 

"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 www.worldkidneyday.org. 

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:
www.alexionpharma.com. 

[ALXN-G] 

References

 1.    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 al., editors. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 1993-. Available from: http://www.ncbi.nlm.nih.gov/books/NBK1367/                                                                                                                                                                                          
 2.    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. 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.                                                                                                                                                                                                                                                                                                                                                     
 4.    Definition from the UK National Institute for Clinical Effectiveness (NICE). 2004. Citizen Council Report on Ultra-Orphan Drugs. Available at http://tinyurl.com/b3qurp3 and as defined in the following documents: 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.                                                                                                                                                                                                                                                                                                                                                                                      
 6.    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.                                                                                                                                                                                                                                                                                                                    
 7.    Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int. 2006;70(1):16-23.                                                                                                                                                                                                                                                                                                                                                                                                    
 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, 203-272-259
Executive Director, Corporate Communications
or
Kim Diamond, 203-439-9600 (media)
Director, Corporate Communications 

Copyright Business Wire 2013

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