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 to ALN-AT3 as a therapeutic 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).
“a major scientific breakthrough that happens once every decade or so”
"We are very pleased to have received Orphan Drug Designation from the FDA for ALN-AT3, a key program in our 'Alnylam 5x15' product development and commercialization strategy. We believe that our subcutaneously delivered RNAi therapeutic represents an innovative approach for the management of hemophilia and has great potential to make a meaningful impact in the treatment of this often debilitating bleeding disorder," said Saraswathy (Sara) Nochur, Ph.D., Senior Vice President, Regulatory Affairs and Quality Assurance at Alnylam. "We look forward to advancing this important program towards the clinic in the months to come."