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Protagonist Therapeutics Announces U.S. FDA Approval of Hepcidin Mimetic Peptide MIMRYLO(TM) (rusfertide) for Polycythemia Vera

PTGX
  • Takeda received FDA approval for MIMRYLO (rusfertide), the first and only hepcidin mimetic peptide for treating erythrocytosis in adults with polycythemia vera (PV).
  • The approval was supported by Phase 3 VERIFY study results showing 76.9% of patients achieved a response during weeks 20-32.
  • Protagonist Therapeutics received $275 million in immediate milestone payments, consisting of a $200 million opt-out fee and a $75 million approval milestone.
  • Protagonist is eligible for up to an additional $875 million in potential future milestones and tiered royalties ranging from 14% to 29% on worldwide net sales.
  • MIMRYLO functions by mimicking hepcidin to control iron availability, thereby suppressing red blood cell production and helping restore iron balance.
  • Clinical benefits include meaningful reduction in phlebotomy burden, improved hematocrit control, and reduced fatigue as measured by PROMIS Fatigue Short Form 8a.
  • MIMRYLO is administered as a weekly subcutaneous injection and is currently approved in the U.S. for the treatment of erythrocytosis in adults with PV.
  • The most common adverse reactions (occurring in >15% of patients) were injection site reactions (56%) and anemia (16%).
  • Warnings include the risk of new or worsening thrombocytosis, injection site reactions, embryo-fetal toxicity, and the recommendation to avoid breastfeeding during treatment.
  • Takeda will commercialize MIMRYLO globally under a license agreement entered into in 2024, having assumed responsibility for NDA submission after Protagonist's opt-out in April 2026.
  • Protagonist Therapeutics held an investor call on August 31st to discuss the approval, marking its second FDA approval of 2026 following ICOTYDE.