The European Medicines Agency (EMA) has granted orphan drug designation to PTG-300 for the treatment of polycythemia vera (PV). PTG-300 is an injectable synthetic hepcidin mimetic peptide that is currently being developed for the treatment of PV and hereditary hemochromatosis.1
In June 2020, PTG-300 was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of PV.
PV is a type of myeloproliferative neoplasm causing elevated levels of red blood cells, which may lead to thrombotic events. Maintaining hematocrit levels below 45% may help reduce the risk of thrombotic events, and PTG-300 provides a non-cytoreductive mechanism targeting iron regulation to control hematocrit levels. Targeting hepcidin may also improve symptoms and systemic iron deficiency in patients with PV.
A phase II study is currently enrolling, and there are ongoing discussions for a pivotal study with PTG-300 in patients with PV, expected to begin in mid-2021.1