CAMBRIDGE, Mass., Aug 14, 2013 (AP) — The Food and Drug Administration has granted an “orphan drug designation” to a potential hemophilia treatment from Alnylam Pharmaceuticals Inc.
Orphan drug status is awarded to drugs that could treat diseases that affect fewer than 200,000 people in the United States. It comes with some added marketing exclusivity.
The Cambridge, Mass., company said Wednesday that the agency gave the designation to a drug labeled ALN-AT3 for the treatment of hemophilia B. Alnylam has tested the drug in mice and plans to start studies involving humans early next year.
for the treatment of hemophilia B. Alnylam has tested the drug in mice and plans to start studies involving humans early next year.
http://www.pharmalive.com/alnylam-hemophilia-drug-garners-orphan-drug-status
My Blog on Medicinal Chemistry 