Polycythemia vera is a myeloproliferative, potentially fatal disease which manifests itself in the form of an overabundance of blood cells and platelets in the body resulting in heart failure, thrombosis, heart attack and stroke.
MGN-4893 targets microRNA-451, a recently discovered key regulator of blood cell development believed to be responsible for many diseases.
miRagen Therapeutic president and CEO William Marshall said the FDA granting the orphan drug status to MGN-4893 for the treatment of polycythemia vera raises hopes of advancing the compound into human clinical trials.
Currently, miRagen is conducting pre-clinical studies of MGN-4893.
The company expects to initiate human clinical trials in 2012.