Heart & blood Hepcidin mimetic peptide
Rusfertide
Regulatory status
Status at a glance
503A compounding status
Not nominated
We found no pending nomination for the 503A bulks list. Unless the substance has another legal pathway (for example, a USP monograph or use in an FDA-approved drug), 503A pharmacies generally may not compound with it.
- FDA approval
- An FDA-approved product contains this peptide
- 503B outsourcing
- Not determined
- Anti-doping (WADA)
- Not on the WADA Prohibited List
- DEA schedule
- Not a controlled substance
About peptide status
Most peptides discussed here are not FDA-approved for any use, and products labeled “research use only” are not for human use. Removal from Category 2 does not authorize compounding. PCAC votes are non-binding recommendations; FDA and HHS make the final decisions.
This is not medical or legal advice, and we do not publish dosing, sourcing or reconstitution information. Talk with a licensed clinician before using any peptide.
If you think you are having a medical emergency, call 911.
Key takeaway
Rusfertide is a hepcidin-mimetic peptide that limits the body's iron supply for red-blood-cell production, developed by Protagonist Therapeutics and Takeda for polycythemia vera, a blood disorder of overproduced red cells. In Phase 2 and Phase 3 trials it controlled hematocrit and reduced the need for therapeutic blood draws. It was approved by the FDA in 2026 under the brand name Mimrylo.
At a glance
- What it is
- A hepcidin-mimetic peptide, sold as Mimrylo
- Approved for
- Polycythemia vera in adults, as a first-in-class treatment
- Key fact
- In trials it controlled hematocrit and reduced the need for phlebotomy
- Route
- Subcutaneous injection
- Evidence strength
- FDA approved
Summary
Rusfertide is an FDA-approved hepcidin-mimetic peptide for polycythemia vera. Learn how it limits iron for red blood cells and what the trials showed.
What is rusfertide?
Rusfertide is a peptide that imitates hepcidin, the body's master hormone for iron regulation. It is used to treat polycythemia vera, a myeloproliferative disorder in which the bone marrow makes too many red blood cells, thickening the blood and raising the risk of clots. It is developed by Protagonist Therapeutics and Takeda and is given by subcutaneous injection under the brand name Mimrylo.
How does rusfertide work?
Rusfertide mimics hepcidin by binding ferroportin, the channel that exports iron from gut cells and from the body's iron stores. By limiting that export, it reduces the iron available for the bone marrow to build new red blood cells. Because red-cell production depends on a steady iron supply, restricting iron slows that production and helps keep hematocrit, the proportion of blood made up of red cells, under control, reducing the need for repeated therapeutic blood draws (phlebotomy).
Is it FDA-approved?
Editor’s note
Status: Yes. The FDA approved rusfertide on August 28, 2026, as Mimrylo for adults with polycythemia vera, a first-in-class approval for a hepcidin-mimetic peptide, based on the Phase 3 VERIFY trial.
Category: In clinical trials · Primary body area: Heart & blood
Yes. The FDA approved rusfertide on August 28, 2026, as Mimrylo for adults with polycythemia vera, a first-in-class approval for a hepcidin-mimetic peptide, based on the Phase 3 VERIFY trial.
What is rusfertide prescribed for?
Rusfertide is prescribed for adults with polycythemia vera to help control hematocrit and reduce reliance on phlebotomy, the standard approach of removing blood to lower red-cell counts. It is used under the direction of a hematologist as part of managing the disease and its clotting risk.
What are the side effects?
According to the prescribing information, the most common side effects are:
- Injection-site reactions
- Anemia
More serious considerations include:
- New or worsening thrombocytosis (a high platelet count)
- Harm to an unborn baby
Sources: the REVIVE Phase 2 trial (Kremyanskaya et al., New England Journal of Medicine, 2024, DOI: 10.1056/NEJMoa2308809) and an open-label Phase 2 study (Chew et al., Leukemia Research, 2025, DOI: 10.1016/j.leukres.2025.108132), both indexed on PubMed; and FDA prescribing information for Mimrylo. This document is educational and does not constitute medical advice.
Registered human trials
0 registered human trials on ClinicalTrials.gov that we reviewed
Sources
- [1]
- [2]
- [3]
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