The U.S. Food and Drug Administration (FDA) gave orphan drug designation to ARCA biopharm, Inc.'s rNAPc2 compound as a potential treatment for Ebola on Wednesday.
ARCA biopharm specializes in genetically targeted therapies for cardiovascular conditions and diseases. rNAPc2 started as a treatment option for deep vein thrombosis and other cardiovascular diseases. In this form, it performed exceptionally well in clinical trials on more than 700 patients.
As a treatment for hemorrhagic fevers, it performed effectively in non-human primates infected with two types of hemorrhagic fever virus -- Ebola and Marburg.
The compound acts as an inhibitor of tissue factors responsible for hemorraghing, an often fatal complication of Ebola. The results of abnormal coagulation tissue factors, systemic consumption coagulopathy and related bouts of inflammation are partially to blame for the high mortality of hemorrhagic fever deaths.
Orphan drug designation from the FDA indicates that the compound in question treats a disease that is rare, affecting fewer than 200,000 individuals in the U.S. This allows the developers to market the product, and develop financing options to further develop and enhance the compound with a period of exclusivity lasting seven years.