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Protagonist Therapeutics Receives FDA Breakthrough Therapy Designation for Rusfertide in Polycythemia Vera
Designation further validates registrational path for rusfertide in polycythemia vera and facilitates potentially expedited development and review NEWARK,
About this update from Protagonist Therapeutics, Inc.
[{"type":"text","content":"Designation further validates registrational path for rusfertide in polycythemia vera and facilitates potentially expedited development and review\n\n\nNEWARK, Calif., June 3, 2021 /PRNewswire/ -- Protagonist Therapeutics (\"Protagonist\" or \"the Company\") (Nasdaq: PTGX), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its lead investigational new drug candidate, rusfertide, for the treatment of patients with polycythemia vera (PV) for the reduction of erythrocytosis in those patients who do not require further treatment for thrombocytosis and/or leukocytosis. Breakthrough Therapy Designation requires that the drug candidate treat a serious or life-threatening disease or condition. It also requires preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The designation has the potential to expedite the development and regulatory review process. \n\"We are thrilled to receive the Breakthrough Therapy Designation for rusfertide in PV, a serious disease where the need for different and better treatment options is clear and pressing,\" said Suneel Gupta, PhD, Chief Development Officer at Protagonist. \"Rusfertide is a natural hormone mimetic and may stand out as the first non-cytoreductive therapeutic drug for PV. We look forward to working closely with FDA regulators to advance and complete all relevant clinical studies, both ongoing and planned, as quickly as possible.\"\nThe designation for rusfertide was supported in part by promising data from the ongoing Phase 2 clinical trial in patients with PV, presented at the Annual Meeting of the American Society of Hematology in 2020. The data showed that when treated with rusfertide, a majority of patients were able to eliminate therapeutic phlebotomies, maintain a target hematocrit level of less than 45 percent, reverse iron deficiency, and experience symptom improvements. The FDA previously granted orphan drug status and Fast Track Designation to rusfertide in PV. Breakthrough Therapy Designation offers additional advantages over Fast Track Designation, including FDA actions to expedite both planned clinical trials and plans for expediting the manufacturing development strategy. \nUpdated data from the ...