FDA APPROVES ALEXION'S SOLIRIS FOR PNH

Alexion Pharmaceuticals announced it has received marketing approval from the FDA for Soliris (eculizumab). Soliris is the first therapy approved for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder defined by chronic red blood cell destruction, or hemolysis.

Patients with PNH are missing a specific protein that normally protects red blood cells from destruction by a component of the immune system called terminal complement. Soliris, the first complement inhibitor approved in the U.S., prevents hemolysis by selectively blocking terminal complement.

Soliris has proven to be a safe and effective therapy for PNH in three multinational clinical studies: TRIUMPH, a placebo-controlled, 26-week, Phase III study involving 87 PNH patients; SHEPHERD, an open-label, 52-week, Phase III trial involving 97 PNH patients; and E05-001, a long-term extension study.

These studies showed that Soliris reduced hemolysis in every treated patient. The reductions in hemolysis occurred within one week of initiating treatment and were sustained for periods of up to 54 months with continued dosing of Soliris. The reduction in hemolysis expands the number of circulating PNH cells and, thereby, increases the hemoglobin level. Hemoglobin stabilization and the number of transfused packed red blood cell units, the pivotal study's co-primary endpoints, were both achieved. Also, Soliris patients reported less fatigue and improved health-related quality of life. There were fewer thrombotic events with Soliris treatment than during the same period of time prior to treatment.