Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced the presentation of two-year data that highlight the long-term benefits of chronic Soliris ® (eculizumab) therapy in patients with atypical hemolytic uremic syndrome (aHUS), an ultra-rare genetic disease characterized by thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body. In two pivotal phase 2 studies, which initially enrolled 37 patients, 32 patients entered a long-term extension phase. The data demonstrated that ongoing Soliris treatment for two years was associated with sustained inhibition of complement-mediated TMA, as indicated by a maintained increase in platelet count, and sustained improvement in renal function and TMA event-free status. 1,2 The data were presented today at Kidney Week 2012, the annual meeting of the American Society of Nephrology (ASN) in San Diego. Additionally, data presented in a poster session at ASN support early initiation of Soliris therapy regardless of the presence or absence of genetic mutations in patients with aHUS. 3
aHUS is an ultra-rare, life-threatening, chronic genetic disease that can progressively damage vital organs, leading to stroke, heart attack, kidney failure, and death. 4 The morbidity and premature mortality in aHUS is caused by chronic uncontrolled activation of the complement system, resulting in TMA. 5,6 Soliris, a first-in-class terminal complement inhibitor, specifically targets uncontrolled complement activation, and is approved for the treatment of patients with aHUS to inhibit complement-mediated TMA.
“These long-term extension studies show that early and chronic treatment with Soliris leads to continued improvement in patient outcomes for up to two years, as demonstrated by continued inhibition of complement-mediated TMA, and improvement in renal function,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “Additional data presented at ASN show that clinicians need not wait for genetic mutation analyses before initiating Soliris therapy, further supporting the rationale for commencing Soliris therapy at the time of clinical diagnosis of aHUS.”
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