Canada-based drug maker YM BioSciences (TSE:YM) said on Tuesday that it has received orphan drug designation from the European Commission for CYT387, a treatment for myelofibrosis, a type of chronic leukemia.
Myelofibrosis is a serious bone marrow disorder that disrupts the body’s normal production of blood cells, as bone marrow is replaced with scar tissue. The result is extensive scarring in the bone marrow, leading to severe anemia, weakness, fatigue, and often, an enlarged spleen and liver.
The European Commission grants orphan designation to medicines for the treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union.
Developers receiving orphan designation status may be given fee reductions, protocol assistance, access to centralized authorization procedures, and 10 years of marketing exclusivity once a drug is authorized.
Currently, CYT387 is in phase one and two trials for myelofibrosis.
“Having CYT387 designated as an orphan drug for the treatment of myelofibrosis in Europe, in addition to our previously obtained orphan designation in the US, will help ensure that we are able to advance this drug as efficiently as possible for these patient groups,” said president and CEO Nick Glover.
Indeed, CYT387 received orphan drug designation from the US FDA last August.
CYT387 is an oral drug that inhibits JAK1 and JAK2 kinases, enzymes which have been implicated in many immune cell disorders including cancers like prostate, breast, head and neck and lung.
YM BioSciences is currently advancing three clincial stage products, including CYT387, and holds a library of more than 4,000 compounds that are in the process of being evaluated.–Brad Lemaire