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U.S. FDA grants orphan drug designation for PTG-300 for the treatment of polycythemia vera

Jun 23, 2020

PTG-300, an injectable hepcidin mimetic peptide, has received orphan drug designation by the U.S. Food and Drug Administration on June 12, 20201.

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 PV2. 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-level2.

  1. U.S. Food and Drug Administration. Orphan drug designations and approvals. June 19, 2020. Accessed June 19, 2020.
  2. Cision PR Newswire. Hepcidin mimetic PTG-300 receives U.S. FDA orphan drug designation for the treatment of polycythemia vera. Published June 17, 2020. Accessed June 19, 2020.