Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN), today announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has approved the use of Soliris® (eculizumab) for the treatment of pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS), a life-threatening ultra-rare disorder. Soliris is already approved in Japan for paroxysmal nocturnal hemoglobinuria (PNH), another severe and ultra-rare disease. Alexion expects that initial patients with aHUS in Japan will commence treatment with Soliris during the fourth quarter of this year.
aHUS is a life-threatening and ultra-rare chronic genetic disease that can progressively damage vital organs, leading to stroke, heart attack, kidney failure, and death.
The morbidities and premature mortality in aHUS is caused by chronic uncontrolled activation of the complement system, resulting in thromobotic microangiopathy (TMA, the formation of blood clots in small blood vessels throughout the body).
Soliris, a first-in-class terminal complement inhibitor, specifically targets uncontrolled complement activation, and is approved for the suppression of TMA in patients with aHUS.
"Soliris has been proven to have a life-transforming impact on patients suffering from aHUS and represents a significant step forward in the treatment of this devastating disease," said Prof. Motoshi Hattori, Professor and Director of the Department of Pediatric Nephrology, Tokyo Women’s Medical University of Tokyo Woman’s UHP. "Results from clinical studies show that chronic Soliris treatment inhibits complement-mediated TMA which is responsible for thrombosis, vital organ failure, and other life-threatening manifestations of aHUS.”
"The approval of Soliris for the treatment of aHUS by the Japanese government brings life-transforming hope to patients suffering from this devastating disease," said David Hallal, Executive Vice President and Chief Commercial Officer of Alexion. "We will initiate our disease awareness and education programs as we work closely with the healthcare community to support rapid and accurate diagnosis of aHUS as well as better informed treatment decisions."