RNAi therapeutics company Alnylam Pharmaceuticals has obtained additional orphan drug designation from US FDA for a RNAi therapeutic, ALN-AT3, to treat hemophilia A.
With the additional approval, orphan drug designation for GalNAc conjugate delivery platform-based ALN-AT3 now includes treatment of both hemophilia A and hemophilia B.
The subcutaneous RNAi therapeutic targeting antithrombin (AT) is being developed for the treatment of hemophilia, including hemophilia A, hemophilia B, and hemophilia A or B with inhibitors, and other rare bleeding disorders (RBD).
Alnylam regulatory affairs and quality assurance senior vice president Dr Saraswathy Nochur said the FDA has granted orphan drug designation for ALN-AT3 now for both the treatment of hemophilia A and hemophilia B.
"As a subcutaneously delivered RNAi therapeutic, we believe it 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," Nochur added.
"ALN-AT3 is a key program in our 'Alnylam 5x15' product development and commercialization strategy, and we look forward to advancing this promising RNAi therapeutic into the clinic in the months to come."
Pre-clinical data demonstrated the ability of ALN-AT3 to normalize thrombin generation and improve hemostasis in hemophilia mice and to fully correct thrombin generation in a non-human primate (NHP) hemophilia inhibitor model.