Alnylam Pharma Gets Addl. Orphan Drug Designation For ALN-AT3 For Hemophilia A

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY), a RNAi therapeutics company, said the U.S. Food & Drug Administration or FDA has granted an Orphan Drug Designation or ODD to ALN-AT3 as a therapeutic for hemophilia A treatment. Earlier, 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 or AT, for the treatment of hemophilia, including hemophilia A, hemophilia B, and hemophilia A or B with "inhibitors", and other Rare Bleeding Disorders or RBD.

ALN-AT3 makes use of the firm's proprietary GalNAc conjugate delivery platform, enabling subcutaneous dose administration. Alnylam plans to file an investigational new drug or IND application for ALN-AT3 in the fourth quarter of 2013 and initiate a Phase I study in early 2014.

The FDA Office of Orphan Products Development or OOPD mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.