ALN-AT3 Granted Orphan Drug Designation for Hemophilia A

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

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).

The FDA previously granted ALN-AT3 orphan drug designation for the treatment of hemophilia B.

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