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Mar 19, 2007

First Treatment for PNH Approved

  • Alexion Pharmaceuticals received marketing approval for Soliris™ (eculizumab). Soliris is the first therapy approved for paroxysmal nocturnal hemoglobinuria (PNH), according to the company.

    "Soliris directly targets the underlying disease process responsible for debilitating symptoms that may contribute to shortened life spans of PNH patients," remarks Wendell F. Rosse, M.D., Florence McAlister professor of medicine emeritus, Duke University and an Alexion consultant. "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."

    Soliris is indicated for the treatment of patients with PNH to reduce hemolysis. 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, reportedly the first complement inhibitor approved in the U.S. for the treatment of any disease, prevents hemolysis by selectively blocking terminal complement.

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