Rusfertide

An FDA-approved hepcidin mimetic sold as Mimrylo for treatment of erythrocytosis in adults with polycythemia vera. First-in-class therapy that targets iron availability for erythropoiesis. Research-chemical or compounded listings are not the labelled Mimrylo product.

Rusfertide is a peptide mimetic of the endogenous hormone hepcidin. Per the Mimrylo label, it blocks the iron transporter ferroportin. Inhibition of ferroportin reduces iron available for red-blood-cell production and lowers hematocrit. That is the labelled mechanism for the finished Mimrylo product, not a protocol.

VERIFY (NCT05210790) is the Phase 3 multicenter, randomized, double-blind, placebo-controlled study cited on the Mimrylo label and in the FDA 28 Aug 2026 press announcement: 293 adults with PV who required frequent phlebotomies despite standard-of-care therapy; randomized 1:1 to Mimrylo or placebo over 32 weeks. Per FDA press, 76.9% on Mimrylo versus 32.9% on placebo required no phlebotomies during the randomized period (efficacy measured as proportion not meeting phlebotomy criteria between weeks 20 and 32). ClinicalTrials.gov lists enrollment n=293 actual; results section not posted on CT.gov at draft time. No PubMed hit for a VERIFY primary manuscript was found. Peer-reviewed Phase 2 REVIVE (Kremyanskaya et al., N Engl J Med 2024; PMID 38381675; DOI 10.1056/NEJMoa2308809; NCT04057040) reported hematocrit control and phlebotomy reduction in a smaller randomized withdrawal design (not the pivotal VERIFY population). Evidence grade is Moderate: approved label + FDA announcement + registry for VERIFY, plus earlier peer-reviewed Phase 2, without a peer-reviewed VERIFY paper yet.

• FDA press announcement — Mimrylo (rusfertide) approval · 2026

  FDA Approves First Drug of Its Kind for Polycythemia Vera. Immediate release 28 Aug 2026. VERIFY n=293 Phase 3 summary; first-in-class hepcidin mimetic.

• Mimrylo (rusfertide) prescribing information (DailyMed) · 2026

  SPL setid 101aff01-31ed-4841-a8b6-0a5a8503189b. Indication: erythrocytosis in adults with PV. Section 4 contraindications: none. Mechanism: hepcidin mimetic / ferroportin blockade. Clinical studies: VERIFY NCT05210790.

• VERIFY — ClinicalTrials.gov NCT05210790

  Phase 3 study of rusfertide (PTG-300) in polycythemia vera. Enrollment n=293 actual. Primary completion listed; peer-reviewed primary manuscript not identified on PubMed at draft time.

• Kremyanskaya et al., REVIVE (Phase 2) · 2024 · N Engl J Med

  Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera. N Engl J Med. 2024. PMID 38381675. DOI 10.1056/NEJMoa2308809. NCT04057040. Phase 2 randomized withdrawal; not VERIFY. PMID 38381675.