Alnylam Pharmaceuticals announced that the U.S. Food & Drug Administrationhas granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the treatment of hemophilia B. Alnylam is developing ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin for the treatment of hemophilia, including hemophilia A, hemophilia B, and hemophilia A or B with “inhibitors," and other Rare Bleeding Disorders. Alnylam plans to file an investigational new drug application for ALN-AT3 in Q4 and initiate a Phase I clinical trial in early 2014.
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