Rusfertide

Rusfertide Receives Breakthrough Therapy Designation for Treatment of Erythrocytosis in Patients with Polycythemia Vera

NEWARK, CA -  Protagonist Therapeutics, Inc.  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. Breakthrough Therapy Designation also conveys eligibility for priority NDA review and Orphan Drug Designation qualifies sponsors for various incentives including a potential for extended market exclusivity.

Breakthrough 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.

Rusfertide is being co-developed with Takeda Pharmaceuticals pursuant to a worldwide collaboration and license agreement entered in 2024, under which Protagonist remains primarily responsible for development through NDA filing.

Breakthrough Therapy designation complements Rusfertide’s Orphan Drug and Fast Track designations, which together confer multiple benefits to the development program.

U.S. New Drug Application filing for Rusfertide in polycythemia vera on track for Q4.