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

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.

“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.”

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.^4,6Sixty-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.

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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.