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Eculizumab Receives Orphan Drug Designation in the U.S. and Europe
CHESHIRE, Conn., Dec. 8 /PRNewswire-FirstCall/ -- Alexion Pharmaceuticals Inc. announced today that both the U.S. Food & Drug Administration and the European Agency for the Evaluation of Medicinal Products have granted eculizumab orphan drug status for the treatment of PNH.
"The assignment of orphan drug status for eculizumab in PNH in both the U.S. and European Union is an important element of our global development and commercialization plan for eculizumab in this underserved population," stated Leonard Bell, M.D., Chief Executive Officer of Alexion Pharmaceuticals, Inc. "We are currently discussing our PNH program with the FDA so that we can finalize plans to advance development of eculizumab in PNH at the soonest opportunity."
Orphan Drug Designation allows special incentives for sponsors planning to test a product for use in a rare disease or condition. These incentives include eligibility for research grants, certain tax benefits, protocol assistance, and possible exemptions or reductions of certain regulatory fees during development or at the time of application for marketing approval. Once approved, the product may qualify for seven years of marketing exclusivity in the U.S. and 10 years of marketing exclusivity in the European Union.
Patients with PNH have a deficiency in certain protective proteins on the surface of their red blood cells, allowing their own complement system to attack and destroy these cells, resulting in hemolysis. Depending on the severity of this hemolysis, patients may become anemic and experience significant fatigue. Severe anemia is debilitating and has been shown to lead to a marked impairment of quality of life. Other severe complications of PNH include deficiencies of other blood cell types and inappropriate blood clotting known as thrombosis, both of which can cause death in these patients. Treatment for PNH is currently limited to corticosteroids, immunosuppressive drugs and blood transfusions. Each has significant side effects and little or no effect on the degree of hemolysis that occurs in PNH. PNH is a rare, chronic disease, with an estimated incidence of 2-6 people per million. Estimated survival of PNH patients after diagnosis is 50% in 10 years and 28% in 25 years.
Source: Alexion Pharmaceuticals Inc.