Soliris is approved for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder defined by the destruction of red blood cells, or hemolysis. In patients with PNH, hemolysis can cause severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark colored urine (hemoglobinuria), kidney disease, impaired quality of life and thromboses.
The study, published online in Blood, the journal of the American Society of Hematology, analyzed data from 195 patients studied in the Soliris Phase II and Phase III trials. Patients were enrolled in the US, Europe, Australia and Canada and treated with Soliris for up to 54 months.
Soliris reduced hemolysis in all treated patients and reduced thrombosis by 92%, with three events during Soliris treatment compared to 39 events during the same period of time prior to treatment. The collection of thrombotic events was defined prospectively by clinical trial protocols.