ALN-AT3 Gains Orphan Drug Status for Hemophilia B

Alnylam Pharmaceuticals announced that the FDA has granted ALN-AT3 Orphan Drug designation for the treatment of hemophilia B.

Alnylam presented pre-clinical data demonstrating that ALN-AT3 can normalize thrombin generation and improve hemostasis in hemophilia mice and can fully correct thrombin generation in a non-human primate (NHP) hemophilia “inhibitor” model. ALN-AT3 utilizes the company's proprietary GalNAc conjugate delivery platform, enabling subcutaneous dose administration.

ALN-AT3 is a subcutaneously administered RNAi therapeutic targeting antithrombin (AT)  being developed for the treatment of hemophilia A, hemophilia B, hemophilia A or B with “inhibitors”, and other Rare Bleeding Disorders (RBD).

For more information call (617) 551-8200 or visit www.alnylam.com.