Evidence review: Open Peptide Project (verified against primary PubMed, ClinicalTrials.gov & FDA records) · Last checked 2026-10-27. ← Back to Evidence Library

Rusfertide (Mimrylo, PTG-300): what the evidence says

Name: Rusfertide (Mimrylo, PTG-300)
Published: 2026-10-27

At a glance

What it is: an injectable peptide that mimics hepcidin, the hormone that controls iron. Limiting iron slows red blood cell production in polycythemia vera. FDA-approved as Mimrylo (Takeda) in Aug 2026. ^{R1, R2}

What the evidence says: Human in VERIFY (293 adults with polycythemia vera needing frequent blood removal), 77% responded vs 33% with placebo. ^R3 The phase 2 REVIVE trial showed a similar pattern. ^R4

Biggest risks: label warnings: high platelet counts (thrombocytosis), injection-site reactions and harm to an unborn baby. Injection-site reactions affected 56% of patients. ^R2

Rating


Human Human evidence	Strong: phase 3 RCT (n = 293) plus phase 2 RCT ^{R3, R4}
Animal Animal / lab evidence	Not reviewed
Regulatory	FDA-approved ^R1 · not on FDA's 503A or 503B bulk-substance category lists and not on FDA's compounding safety-risk page ^{R5, R6, R7} · not named by WADA ^R8
Established human dose	Yes, label dose (see Study details)

Evidence verdict: Polycythemia vera: Strong (phase 3 RCT) · Other uses: No human data

This page reports what studies used. It is not dosing advice.

Legend: HUMAN data · ANIMAL / LAB data · UNKNOWN · UNVERIFIED: not yet confirmed against a primary source

About the evidence: placebo-controlled sponsor trials with clear blood endpoints.

What is it?

Related pages: pegcetacoplan, romiplostim.

Does it work?

Human VERIFY: response 77% vs 33%; hematocrit kept under 45% in 63% vs 14%. ^{R3, R9}
Human REVIVE (phase 2): response 60% vs 17% in the randomized withdrawal part. ^{R4, R10}

Is it safe?

Label warnings: thrombocytosis, injection-site reactions, embryo-fetal toxicity. ^R2
Most common: injection-site reactions 56%, anemia 16%. ^R2

Gray-market vials: extra risks

A gray-market vial has no quality control and no guarantee of identity, strength or sterility. We test retail vials blind under the Open Peptide Standard; see /tests/. A passing test covers one batch on one date and does not mean the compound is safe.

Is it legal?

US: FDA-approved, prescription (Mimrylo, approved Aug 28, 2026). ^R1
Compounding: not on FDA's 503A or 503B bulk-substance category lists and not on FDA's compounding safety-risk page. ^{R5, R6, R7}
Sport: not named on WADA's 2026 Prohibited List. ^R8

Study details

These are amounts researchers used, not recommendations.

Study	People / model	Route	Amount used	Follow-up	Reported	Citation
REVIVE 2024	70 adults with PV	SC	Titrated, per study	Randomized withdrawal	Response 60% vs 17%	PMID 38381675
VERIFY 2026	293 adults with PV	SC	Titrated, per label	32 weeks	Response 77% vs 33%	PMID 42868322

Full reference list (PubMed, CT.gov, FDA and WADA checked Oct 27, 2026)

^R1 Drugs@FDA, Mimrylo (rusfertide), application NDA220605 (openFDA drugsfda record checked Oct 27, 2026). https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=NDA220605
^R2 Mimrylo prescribing information, label published Aug 28, 2026 (DailyMed, checked Oct 27, 2026). https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=101aff01-31ed-4841-a8b6-0a5a8503189b
^R3 Kuykendall AT, et al. J Clin Oncol. 2026. PMID 42868322
^R4 Kremyanskaya M, et al. N Engl J Med. 2024. PMID 38381675
^R5 FDA, 503A bulk drug substances categories list (updated May 14, 2026). https://www.fda.gov/media/94155/download
^R6 FDA, 503B bulk drug substances categories list (updated Mar 21, 2025). https://www.fda.gov/media/94164/download
^R7 FDA, Certain bulk drug substances for use in compounding that may present significant safety risks (content current Apr 22, 2026). https://www.fda.gov/drugs/human-drug-compounding/certain-bulk-drug-substances-use-compounding-may-present-significant-safety-risks
^R8 WADA. The 2026 Prohibited List, full list checked; compound not named. https://www.wada-ama.org/sites/default/files/2025-09/2026list_en_final_clean_september_2025.pdf
^R9 ClinicalTrials.gov NCT05210790, VERIFY, phase 3 (CT.gov API v2, checked Oct 27, 2026). https://clinicaltrials.gov/study/NCT05210790
^R10 ClinicalTrials.gov NCT04057040, REVIVE, phase 2, completed, 70 enrolled (CT.gov API v2, checked Oct 27, 2026). https://clinicaltrials.gov/study/NCT04057040

Disclaimer

This page is for education and harm reduction, not medical advice or a recommendation to use Rusfertide (Mimrylo, PTG-300). This is a prescription medicine. Use it only under the care of a licensed clinician; do not buy it from research-chemical or gray-market sellers. Report problems to FDA MedWatch (https://www.fda.gov/safety/medwatch).