The FDA ( Food and Drug Administration ) has approved Soliris ( Eculizumab ).
Soliris is the first therapy approved for paroxysmal nocturnal hemoglobinuria ( PNH ), a rare, disabling and life-threatening blood disorder defined by chronic red blood cell destruction, or hemolysis. Soliris is indicated for the treatment of patients with PNH to reduce hemolysis.
Hemolysis can cause one or more of the following symptoms in patients with paroxysmal nocturnal hemoglobinuria: severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of hemoglobinuria, kidney disease, impaired quality of life and thromboses.
Paroxysmal nocturnal hemoglobinuria often strikes people in the prime of their lives, with an average age of onset in the early 30's.
The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.
Patients with paroxysmal nocturnal hemoglobinuria are missing a specific protein that normally protects red blood cells from destruction by a component of the immune system called terminal complement. Soliris, a complement inhibitor, prevents hemolysis by selectively blocking terminal complement.
Soliris has proven to be a safe and effective therapy for paroxysmal nocturnal hemoglobinuria in three multi-national clinical studies: TRIUMPH, a placebo-controlled 26 week Phase 3 study involving 87 PNH patients, SHEPHERD, an open-label 52 week Phase 3 trial involving 97 PNH patients, and E05-001, a long term extension study.
These studies showed that Soliris reduced hemolysis in every treated patient. Hemolysis was dramatically reduced from a baseline LDH of 2,032 U/L to 239 U/L at week 26 ( p