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Alnylam Hemophilia Drug Gets European Orphan Drug Status


NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week announced that its Phase I hemophilia drug ALN-AT3 has received orphan drug status from European regulators.

ALN-AT3 comprises siRNAs targeting antithrombin, an endogenous inhibitor of thrombin generation. It is delivered subcutaneously using Alnylam's GalNAc conjugation technology.

Earlier this year, Alnylam released data from the first part of a Phase I trial that showed that single 0.03 mg/kg doses of the drug were able to silence its target by up to 28 to 32 percent in healthy volunteers.

Alnylam is currently conducting the second arm of that study, which is testing multiple, escalating doses of ALN-AT3 in 18 patients with moderate to severe hemophilia A or B.

Initial data from the complete trial are set for release later this year.