WASHINGTON, September 3, 2026 — The U.S. Food and Drug Administration (FDA) has approved rusfertide (Mimrylo™) for adults with a rare blood cancer called polycythemia vera. Polycythemia vera causes the body to make too many red blood cells, which thickens the blood and increases the risk of blood clots, stroke and heart attack.
Rusfertide is a first-of-its-kind treatment that mimics a naturally occurring hormone in the body called hepcidin. The hormone regulates iron levels, which in turn helps keep red blood cell levels in check.
"People living with polycythemia vera may need to undergo frequent blood draws, with a removal of upwards of about a pint of blood to address the problem of overproduction of red blood cells," explained Lore Gruenbaum, Ph.D., Chief Scientific Officer of Blood Cancer United. "Rusfertide gives patients another choice: an at-home injection they can give themselves to reduce their reliance on bloodletting."
In a clinical study, 76.9% of patients treated with rusfertide required no bloodletting during the 32-week study period compared to 32.9% of patients who did not receive the treatment.
Foundational science and collaboration build treatment breakthroughs
Foundational work on changes in the iron metabolism and low levels of hepcidin in polycythemia vera was performed in the laboratory of Yelena Ginzburg, M.D., at Icahn School of Medicine at Mount Sinai in New York City.
In 2019, Blood Cancer United, in partnership with the MPN Research Foundation, funded a grant to Dr. Ginzburg to continue this work which expanded the scientific understanding related to hepcidin and the use of hepcidin mimetics such as rusfertide.
"Our approach to research will always include funding basic, disease-focused science, because we know work like Dr. Ginzburg's is essential to more fully understand complex disease biology, which is so important to identify better and safer treatments," says Dr. Gruenbaum. "We also dedicate millions of dollars annually to clinical research to accelerate the final stages of drug development that bring new treatments forward to FDA approval and then out to patients."
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