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. Patients’s overall quality of life, including fatigue, will also be assessed.
When fully enrolled, the open-label study, authorized by Japan’s Pharmaceutical and Medical Device Administration (PMDA), is expected to include approximately 25 patients with PNH at clinical sites throughout Japan. Each PNH patient in the AEGIS study will be dosed with Soliris for 12 weeks after enrollment.
Leonard Bell, CEO of Alexion, said: “After completing enrollment and dosing, and comparing these results in Japanese patients to our earlier PNH studies, we expect to submit a licensing application for Soliris in PNH to the Japanese regulatory authority.”