WASHINGTON, March 20 (UPI) -- The U.S. Food and Drug Administration has approved Soliris (eculizumab), the first drug produced for the treatment of a rare blood disorder.
The disorder, paroxysmal nocturnal hemoglobinuria, or PNH, can lead to disability and premature death. Soliris is classified as an orphan drug -- a pharmaceutical developed to treat fewer than 200,000 people. Orphan status gives the drug's manufacturer a seven-year period in which to exclusively market the compound.
Soliris, is manufactured by Alexion Pharmaceuticals Inc. of Cheshire, Conn.
"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 Dr. Steven Galson, director of the FDA's Center for Drug Evaluation and Research.
PNH, which usually develops in adults, is a disease characterized by red blood cells that develop abnormally Depending upon the severity of the disorder, patients with PNH may experience pain, fatigue, debilitating weakness, blood clots, and life-threatening or fatal strokes, heart attacks and intestinal disease. They often need frequent blood transfusions.
Soliris does not cure PNH, but treats the breakdown of red blood cells.
