FDA grants orphan drug status to potential treatment for hemophilia

Alnylam Pharmaceuticals Inc., a Cambridge company specializing in a gene-silencing technology known as RNAi, said Wednesday that the Food and Drug Administration has granted orphan-drug status to an experimental hemophilia B treatment.

Such a designation gives a company incentives to develop treatments for rare diseases that do not have mass-market potential. For now, Alnylam is calling its hemophilia drug candidate ALN-AT3.


In a statement, Saraswathy Nochur, a senior vice president, said, “We believe that our subcutaneously delivered RNAi therapeutic 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.”

Hemophilias are hereditary disorders caused by genetic deficiencies of blood-clotting factors, resulting in recurrent bleeds into joints, muscles, and major internal organs.

Chris Reidy can be reached at
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