Protagonist receives Breakthrough Therapy Designation for rusfertide

Protagonist Therapeutics (PTGX) announced that rusfertide as been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration for the treatment of erythrocytosis in patients with polycythemia vera. 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 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 Annual Meeting.