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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
Trial updates for rusfertide (PTG-300) in polycythemia vera
For patients with polycythemia vera (PV), the current standard of care is periodic therapeutic phlebotomy (TP) with or without cytoreductive therapy to reduce the risk of thrombosis and...
PROUD-PV phase III results on ropeginterferon alpha-2b for polycythemia vera
Interferon alpha (IFN-a) compounds have long been used for the treatment of myeloproliferative neoplasms (MPN), showing a combination of clinical, molecular, and...
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