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PTG-300 receives orphan drug designation from EMA for the treatment of PV

Oct 23, 2020

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

  1. Cision PR Newswire. Protagonist Therapeutics receives orphan drug designation from the European Medicines Agency for PTG-300 in polycythemia vera. Published Oct 21, 2020. Accessed Oct 22, 2020.