Alexion Submits Applications for Soliris® (Eculizumab) as a Treatment for Patients with Atypical Hemolytic Uremic Syndrome (aHUS) in the United States

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) announced today that the company has submitted marketing applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for Soliris^® (eculizumab) as a treatment for patients with atypical Hemolytic Uremic Syndrome (aHUS).

Both the US and EU filings include the positive data from the two 26-week Phase 2 studies of Soliris as a treatment for adult and adolescent patients with aHUS. Preliminary data from these two studies were presented at the American Society of Nephrology (ASN) annual meeting in November 2010. ^1-3 Primary endpoints in both studies were achieved with statistical significance. aHUS is an ultra-rare, chronic and life-threatening disease in which uncontrolled complement activation causes blood clots in small blood vessels throughout the body (thrombotic microangiopathy, or TMA) leading to kidney failure, stroke, heart attack and death.

“The US and EU regulatory submissions put us one step closer toward accomplishing our goal to transform the lives of patients suffering with aHUS,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “We recognize that patients with aHUS, a life-threatening and ultra-rare disorder, lack adequate treatment options, and we look forward to working with regulatory authorities as they review our applications.”