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FDA grants orphan drug status to Alnylam’s potential treatment for hemophilia

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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 an orphan drug designation to an Alnylam drug candidate designed to treat hemophilia B.

An orphan drug designation provides a company with incentives for developing treatments for rare diseases that make it to the market. For the moment, Alnylam is calling its hemophilia drug candidate ALN-AT3.

In a statement, Saraswathy Nochur, a company senior vice president, said: “We are very pleased to have received Orphan Drug Designation from the FDA for ALN-AT3, a key program in our ‘Alnylam 5x15’ product development and commercialization strategy. 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. We look forward to advancing this important program towards the clinic in the months to come.”

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Hemophilias are hereditary disorders caused by genetic deficiencies of various blood clotting factors, resulting in recurrent bleeds into joints, muscles, and other major internal organs, Alnylam noted in its press release.

According to Alnylam, RNAi, or RNA interference, represents a breakthrough in understanding how genes are turned on and off in cells and a completely new approach to drug discovery and development.

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