Soliris is also approved by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and other regulatory authorities for the treatment of patients with aHUS to inhibit complement-mediated TMA. In addition, Soliris is approved in more than 40 countries, including Japan, for the treatment of patients with PNH, a debilitating, ultra-rare and life-threatening blood disorder.
Alexion’s supplemental new drug application (sNDA) for Soliris in Japan included clinical data from two prospective, controlled, open-label studies in adolescent and adult patients with aHUS, and a third retrospective study in pediatric patients with aHUS, which together included a broad range of aHUS patients in North America and Europe. In these studies, all patients treated with Soliris demonstrated rapid and sustained reduction in terminal complement activity, and chronic administration of Soliris resulted in rapid and sustained reduction in complement-mediated TMA. Soliris was well tolerated in these clinical studies. The most frequently reported adverse events were hypertension, upper respiratory tract infection, and diarrhea. In addition, the sNDA in Japan included data from a retrospective study and from an ongoing prospective study of both pediatric and adult aHUS patients in Japan. Results from these studies demonstrated that the safety and efficacy of Soliris in these Japanese patients with aHUS were consistent with those noted in the clinical studies of Soliris in aHUS patients in North America and Europe.
About aHUSaHUS is a chronic, ultra-rare, and life-threatening disease in which a genetic deficiency in one or more complement regulatory genes causes chronic uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body. 4,5 Permanent, uncontrolled complement activation in aHUS causes a life-long risk for TMA, which leads to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death. 4,6 Sixty-five percent of all patients with aHUS die, require kidney dialysis, or have permanent kidney damage within the first year after diagnosis despite plasma exchange or plasma infusion (PE/PI). 3,7 The majority of patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90% transplant failure rate in these TMA patients. 8 aHUS affects both children and adults. Complement-mediated TMA also causes reduction in platelet count (thrombocytopenia) and red blood cell destruction (hemolysis). While mutations have been identified in at least ten different complement regulatory genes, mutations are not identified in 30-50% of patients with a confirmed diagnosis of aHUS. 2 About Soliris Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is also approved in the US, European Union, 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).