Unique blood disorder drug, Eculizumab

Soliris (eculizumab), an Orphan Drug, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a very rare blood disorder, has been approved by the US Food and Drug Administration. PNH can lead to disability and premature death. Solaris is a new molecular entity containing an active ingredient never previously marketed in the USA.

Steven Galson, M.D., M.P.H., director, Center for Drug Evaluation and Research, FDA, said "This product is important in that it offers a treatment other than blood transfusion that may help this small population of patients who are often very ill. This approval is one of multiple examples of how the orphan products program can benefit the public health with urgently needed products that would otherwise not be commercially available."

PNH is a disease which generally develops during adulthood. The red blood cells develop abnormally. Once these abnormal cells are present in the bloodstream, naturally occurring proteins designed to destroy bacteria and other organisms break these cells down. This leads to darkened urine and causes anemia. Depending on how serious a patient's disorder is, they can experience varying degrees of pain, fatigue and debilitating weakness - they may require frequent blood transfusions, experience blood clots, strokes, heart failure and intestinal disease. Soliris treats the breakdown of red blood cells.

The FDA based its approval on a randomized, double-blind, placebo-controlled clinical study of 87 patients with PNH, as well as some other clinical studies. Half of the 87 patients receiving Soliris had stabilization of blood hemoglobin concentrations, compared with no stabilization among the placebo (control) group - this was over a 26-week period. The trial also found that the patients receiving Soliris required significantly fewer blood transfusions.

Source: www.epsdrugstore.com