Toronto Univ..SUNNYBROOK HOSP. :Paroxysmal Nocturnal Haematuria.

Paid by OHIP: approx $250,000 a year. Soliris® (eculizumab), is a first-in-class terminal complement inhibitor discovered, developed and commercialized by ALEXION pharmaceuticals,Inc.. Soliris is approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH)1, a progressive and life-threatening disease characterized by the excessive destruction of red blood cells (hemolysis).1,2,3 Soliris is the first and only therapy approved for the treatment of PNH to reduce hemolysis. In the United States and European Union, Soliris is also approved for the treatment of patients with atypical hemolytic uremic syndrome (aHUS), an ultra-rare, life-threatening, genetic disease that can progressively damage vital organs, leading to stroke, heart attack, kidney failure and death.4,5 Soliris is the first and only therapy approved for the treatment of aHUS. Specifically, Soliris is indicated for the treatment of patients with aHUS to inhibit complement-mediated thrombotic microangiopathy, or TMA. The effectiveness of Soliris in aHUS is based on the effects on TMA and renal function. Prospective clinical trials in additional patients are ongoing to confirm the benefit of Soliris in patients with aHUS. Soliris is not indicated for the treatment of patients with Shiga toxin E coli-related hemolytic uremic syndrome (STEC-HUS). Soliris works by selectively targeting and blocking the complement cascade—a normal part of the immune system that, when activated inappropriately, plays a role in serious diseases like PNH and aHUS. Soliris has earned some of the pharmaceutical industry’s highest honors for innovation, including the 2008 Prix Galien USA Award for Best Biotechnology Product with broad implications for future biomedical research and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases. To learn more about Soliris, visit www.Soliris.net. Sunnybrook Haematology Dept: Prof. Richard A.WELLS MD(Newfoundland 1985) D.Phil(Oxon) FRCPC (Int.Med & Haematology).