Peptide Graded

Rusfertide

Does Rusfertide work?

Yes, in large controlled trials. The main trial had 293 people.

AStrong human evidenceFDA approvedas of 2026-10-26

Approval rested on the phase 3 VERIFY trial, a randomized, double-blind, placebo-controlled study of 293 adults with phlebotomy-dependent polycythemia vera, preceded by the phase 2 REVIVE trial of 70 patients with a randomized withdrawal phase. Both showed large, consistent reductions in phlebotomy need. Long-term data run to about four years in open-label follow-up; what is missing is evidence on thrombosis and survival. 13

REVIVE was the phase 2 trial. It enrolled 70 patients with polycythemia vera who needed frequent phlebotomies. Dosing was open-label for 28 weeks. Phlebotomy needs fell sharply. Hematocrit stayed below 45 percent in most patients. Then came a randomized withdrawal phase. Researchers assigned 59 patients to continue rusfertide or switch to placebo for 12 weeks. Of those on rusfertide, 60 percent kept their response, versus 17 percent on placebo. Symptom scores for fatigue and concentration improved. 1

VERIFY was the phase 3 trial. It randomized 293 adults to weekly rusfertide or placebo, on top of their current treatment. Between weeks 20 and 32, 77 percent of rusfertide patients needed no phlebotomy, compared with 33 percent on placebo. Rusfertide also did better on hematocrit control, phlebotomy counts and patient-reported fatigue. Open-label follow-up from the earlier trials now runs to about four years. Phlebotomy rates remain far below baseline. 3

Evidence checklist

  • Randomised controlled trial VERIFY phase 3; REVIVE phase 2
  • Human study over 100 people VERIFY, 293 adults
  • More than one research group Article does not say who ran the trials
  • Clinical outcomes, not surrogate markers Phlebotomy need; thrombosis and survival unknown
  • Human safety data beyond case reports About four years of open-label follow-up

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More on Rusfertide

Citations

  1. Kremyanskaya 2024, N Engl J Med: rusfertide, a hepcidin mimetic, for control of erythrocytosis in polycythemia vera (REVIVE) doi.org
  2. Mimrylo (rusfertide) prescribing information drugs.com
  3. ASCO Post, Aug 2026: FDA approves rusfertide for erythrocytosis in polycythemia vera (VERIFY results) ascopost.com

Reference information only, not medical advice. Drawn from the Rusfertide article, fact-checked against the cited sources. No dosing anywhere on this site.