Peptide that mimics hepcidin, the hormone that regulates iron. Approved by the FDA in 2026 for polycythemia vera, a disease in which the bone marrow makes too many red blood cells.
Regulatory status
Approved (FDA / EMA)
Family
Hepcidin mimetic
Scientific evidence
High
Number of findings
1
Where it acts
Heart and blood
Also known as
rusfertide, PTG-300, TAK-121, Mimrylo
Regulatory status
FDA (US): Approved (August 2026, Mimrylo) for adults with polycythemia vera
EMA (EU): Not approved (no authorization in the EU as of October 2026)
Dose
Subcutaneous injection once a week, self-administered and added to usual treatment (FDA approval 2026, Mimrylo). In an open-label study, 40 mg twice a week was used until a hematocrit <45% was reached and then weekly (PMID 41175501).
Safety
The most frequent effects are injection-site reactions (47%), anemia (26%), and fatigue (20%), almost always mild. Requires medical monitoring of hematocrit.
Effect by goal · level of evidence
Fewer phlebotomies and sustained response (phase 2)
reducesModerateRCT (AA)in people
8.7 → 0.6 phlebotomies/year
REVIVE trial with 70 patients: phlebotomies fell from 8.7 to 0.6 per year. In the randomized withdrawal phase, 60% responded with rusfertide vs 17% with placebo.
Mimics hepcidin and reduces the iron available to make red blood cells. This lowers the hematocrit (the proportion of blood taken up by red blood cells) and far fewer phlebotomies are needed.
What the evidence says
In people
Fewer phlebotomies and sustained response (phase 2). REVIVE trial with 70 patients: phlebotomies fell from 8.7 to 0.6 per year. In the randomized withdrawal phase, 60% responded with rusfertide vs 17% with placebo. PubMed 38381675