While solid organ transplantation is the most effective form of therapy for the treatment of patients with end-stage renal disease (ESRD), 5 concern about the consequences of AMR remains a significant obstacle to transplantation, as it results in significant delays for affected patients to access a suitable transplant. Overall, ESRD patients on dialysis have a very high mortality rate since approximately 65% of these patients die within 5 years of commencing dialysis. 6 Additionally, approximately one-third of patients on the kidney transplant waiting list are sensitized to their potential donors 7 and historically, approximately 30% of this highly sensitized population has developed AMR. 2 A therapy that prevents acute AMR is critically important for sensitized patients with ESRD. Currently, there are no approved therapies for the prevention of acute AMR.
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the United States (US), European Union (EU) and other countries as the first and only treatment for aHUS patients. Soliris is indicated to inhibit complement-mediated TMA. Soliris is not indicated for the treatment of patients with Shiga toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). Alexion is evaluating the safety and efficacy of Soliris for the treatment of patients with STEC-HUS.
Soliris also is approved in the US, EU, Japan and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder characterized by complement-mediated hemolysis (destruction of red blood cells). Soliris is indicated to reduce hemolysis.Alexion's breakthrough approach in terminal complement inhibition has received the pharmaceutical industry's highest honors: 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.