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Achillion drug secures second US FDA orphan drug designation

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Achillion drug secures second US FDA orphan drug designation

Achillion Pharmaceuticals Inc. secured the U.S. Food and Drug Administration's orphan drug designation for ACH-4471 to treat a rare, kidney-related disease known as C3 Glomerulopathy.

ACH-4471 previously received the U.S. FDA's orphan drug designation to treat paroxysmal nocturnal hemoglobinuria that is characterized by red blood cell destruction, blood clots and impaired bone marrow function.