PTG-300, an injectable hepcidin mimetic peptide, has received orphan drug designation by the U.S. Food and Drug Administration on June 12, 2020 1.
Polycythemia vera (PV) is a type of myeloproliferative neoplasm, causing elevated levels of red blood cells which may lead to thrombotic events. Hematocrit levels maintained below 45% may help reducing the risk of thrombotic events.
PTG-300 provides a non-cytoreductive mechanism to control hematocrit levels, thereby managing symptoms and improving systemic iron deficiency in patients with PV 2. It is currently being developed for the treatment of PV and hereditary hemochromatosis and initial phase II results showed a well-tolerated safety profile, as well as a robust clinical response and clinically meaningful dose-related control of hematocrit levels at individual patient-level 2.