FDA OKs Drug to Treat Blood Disorder
Monday, March 19th, 2007WASHINGTON (AP) — The first drug to treat a blood disorder that strikes one in a million people won federal approval Friday. Soliris, or eculizumab, treats paroxysmal nocturnal hemoglobinuria or PNH, the Food and Drug Administration said. The rare blood disorder can lead to disability and premature death. The drug is made by Alexion Pharmaceuticals Inc. of Cheshire, Conn.
The drug is not a cure. Instead, it treats the breakdown of abnormally developed red blood cells that are the hallmark of the disease. If left untreated, the body naturally breaks down those cells, causing anemia. Depending on the severity of the disease, patients with PNH may have pain, fatigue and debilitating weakness and require frequent blood transfusions. It also can lead to blood clots and life-threatening or fatal strokes, heart attacks and intestinal disease, the FDA said.
Since Soliris blocks the body’s natural immune system it can increase susceptibility to serious infections, particularly bacterial meningitis, the FDA said.
The drug’s label flags that risk and requires that patients be vaccinated for meningitis before starting treatment.