FDA News

FDA Approves Drug to Treat Rare PNH Blood Disorder

(March 19, 2007)-- Alexion Pharmaceuticals said that it has received marketing approval from the U.S. Food and Drug Administration (FDA) for 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 PNH: severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark colored urine (hemoglobinuria), kidney disease, impaired quality of life and blood clots (thromboses). PNH 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 PNH are missing a specific protein that normally protects red blood cells from destruction by a component of the immune system called terminal complement. Soliris, the first complement inhibitor approved in the United States for the treatment of any disease, prevents hemolysis by selectively blocking terminal complement.

"Soliris brings real hope to people who live daily with the devastating effects of PNH. With the approval of Soliris, we now have a therapy that dramatically improves the lives of patients suffering from this disease. Importantly, all patients with this life-threatening disease will be eligible for treatment," said Leonard Bell, MD, chief executive officer of Alexion Pharmaceuticals.

"Soliris directly targets the underlying disease process responsible for debilitating symptoms that may contribute to shortened life spans of PNH patients," said Wendell F. Rosse, MD, Florence McAlister Professor of Medicine Emeritus, Duke University. "Having cared for more than 300 patients with PNH over my career, I believe this is the most important advance that has been made in the treatment of this disease. Treatment with Soliris markedly decreases the hemolysis responsible for anemia, fatigue, poor patient functioning and blood clots in PNH patients."

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