Alnylam Pharmaceuticals Receives Additional Orphan Drug Designation From FDA for ALN-AT3, an RNAi Therapeutic for the Treatment of Hemophilia

Published: Aug 20, 2013

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) to ALN-AT3 as a therapeutic for the treatment of hemophilia A. As reported last week, the FDA has also granted ODD to ALN-AT3 for the treatment of hemophilia B. Alnylam is developing ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT), for the treatment of hemophilia – including hemophilia A, hemophilia B, and hemophilia A or B with “inhibitors” – and other Rare Bleeding Disorders (RBD).

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