Business
Rusfertide Receives Breakthrough Therapy Designation for Treatment of Erythrocytosis in Patients with Polycythemia Vera
Breakthrough Therapy designation complements Rusfertide's Orphan Drug and Fast Track designations, which together confer multiple benefits to the development
About this update from Protagonist Therapeutics, Inc.
[{"type":"text","content":"Breakthrough Therapy designation complements Rusfertide's Orphan Drug and Fast Track designations, which together confer multiple benefits to the development programU.S. New Drug Application filing for Rusfertide in polycythemia vera on track for Q4 NEWARK, CA / ACCESS Newswire / August 25, 2025 / Protagonist Therapeutics, Inc. (\"Protagonist\" or \"the Company\") announced today that rusfertide, a potential first-in-class hepcidin-mimetic peptide, has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of erythrocytosis in patients with polycythemia vera (PV). In 2020, rusfertide received Orphan Drug status and Fast Track designation. Breakthrough Therapy Designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies.1 Breakthrough Therapy Designation also conveys eligibility for priority NDA review1 and Orphan Drug Designation qualifies sponsors for various incentives including a potential for extended market exclusivity.2Breakthrough designation for rusfertide was supported by positive 32-week data from the Phase 3 VERIFY study, which was presented as a late breaking abstract highlighting its practice-changing potential during the plenary session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting.\"We are very pleased with the FDA's decision to grant Breakthrough Therapy Designation to rusfertide, which underscores its potential to demonstrate substantial improvement over available PV therapies,\" said Dinesh V. Patel, Ph.D., President and Chief Executive Officer at Protagonist. We remain on track for NDA submission of rusfertide in polycythemia vera by the end of this year.\"\"In the VERIFY Phase 3 trial, rusfertide demonstrated positive results across all primary and key secondary endpoints including hematocrit control, decreased phlebotomy dependence, and patient reported outcomes including improvement in fatigue,\" said Arturo Molina, M.D., M.S., Chief Medical Officer at Protagonist. \"The comprehensive data provide compelling evidence of the potential for rusfertide to address unmet medical needs in patients with PV who are unable to achieve adequate hemato...