Alnylam Pharmaceuticals this week announced that it has received an orphan drug designation from the US Food and Drug Administration for its preclinical siRNA-based drug ALN-AT3 as a treatment for hemophilia A.
Last week, the company said that the drug received an orphan drug designation from the FDA for hemophilia B.
The designation provides a drugmaker with, among other things, extended market exclusivity for its product, tax credits, and marketing incentives.
ALN-AT3 is expected to enter phase I testing next year.