The open-label study, which was authorized by the Japan’s Pharmaceutical and Medical Device Administration (PMDA), over-enrolled a total of 29 patients. The patients will be treated with Soliris at clinical sites throughout Japan for 12 weeks. The primary efficacy endpoint of the study is reduction of hemolysis from baseline. The study will also measure the effect of Soliris on other clinical manifestations of paroxysmal nocturnal hemoglobinuria (PNH), including blood transfusion requirements, thromboses (blood clots), and kidney function.
Leonard Bell, CEO of Alexion, said: “We look forward to completing this trial later this year, and then including the results in our application for Soliris marketing authorization in Japan. One year after the first approval for Soliris, in the US, we remain committed to our goal that every patient with PNH who can benefit from Soliris will have access to it.”