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FDA Approves First-of-its-Kind Drug to Treat Rare Blood Disorder

The U.S. Food and Drug Administration (FDA) today approved Soliris (eculizumab), the first product for the treatment of paroxysmal nocturnal hemoglobinuria// (PNH), a rare type of blood disorder that can lead to disability and premature death. Soliris is classified as an Orphan Drug and is a new molecular entity containing an ingredient not previously marketed in the United States.

"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," said Steven Galson, M.D., M.P.H., director, Center for Drug Evaluation and Research, FDA. "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, which usually develops in adults, is a disease characterized by red blood cells that develop abnormally. Once the abnormal cells are present in the bloodstream, naturally occurring proteins (called the complement system) designed to destroy bacteria and other infection-causing organisms break these cells down. This leads to abnormally darkened urine and, more importantly, causes anemia. Depending upon the severity of the disorder, patients with PNH may have pain, fatigue and debilitating weakness, the need for frequent blood transfusions, blood clots, and life-threatening or fatal strokes, heart attacks and intestinal disease.

Soliris does not cure PNH, but treats the breakdown of red blood cells, the most common characteristic of PNH. Soliris acts to block the complement system activity, including the destruction of PNH red blood cells.

FDA based its approval on the company's randomized, double-blind, placebo-controlled clinical study of 87 patients with PNH and a series of other clinical studies. The controlled study showed that over a 26-week period half of the participants receiving Soliris had stab
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