Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) today announced that researchers presented data from four clinical trials, all demonstrating the clinical benefits of Soliris ® (eculizumab) for the treatment of atypical hemolytic uremic syndrome (aHUS), a genetic, chronic, ultra-rare disease associated with vital organ failure and premature death. Soliris is the first and only approved safe and effective treatment for pediatric and adult patients with aHUS. In two large, prospective, multinational studies, Soliris inhibited systemic complement-mediated thrombotic microangiopathy (TMA, the formation of blood clots in small blood vessels throughout the body) and improved renal function in both pediatric and adult patients with aHUS. 1,2 The data were presented at Kidney Week 2013, the annual meeting of the American Society of Nephrology (ASN) in Atlanta.
The ASN meeting also featured the presentation of three-year update data from two pivotal Phase 2 extension studies that highlighted the long-term benefits of Soliris therapy in patients with aHUS. In these studies, ongoing Soliris treatment at the three-year update was associated with sustained inhibition of complement-mediated TMA, as indicated by stabilization or continued improvement in key hematologic and renal endpoints, and quality of life. 3,4 Additionally, investigators presented initial characteristics from patients enrolled in a global aHUS Registry, which is prospectively collecting information to enhance understanding of the disease process in order to help optimize care and improve quality of life for patients with aHUS. 5
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. 6 The morbidities and premature mortality in aHUS are caused by chronic, uncontrolled activation of the complement system, resulting in systemicTMA. 7,8 Soliris, a first-in-class terminal complement inhibitor, specifically targets uncontrolled complement activation, and is the first and only approved treatment for patients with aHUS in the United States, European Union, Japan and other countries.
“Results from four prospective studies demonstrate a significant and sustained inhibition of complement-mediated TMA with Soliris treatment and support the chronic use of Soliris in pediatric and adult patients with aHUS,” said Leonard Bell, Chief Executive Officer of Alexion. “These studies further underscore the rationale for initiating Soliris therapy at the time of clinical diagnosis of aHUS and chronic treatment of patients to achieve optimal outcomes.”