Chemistry:Rusfertide
Rusfertide is an investigational new drug that is being developed by Protagonist Therapeutics in partnership with Takeda for the treatment of polycythemia vera (PV).[1] It is a cyclic peptide that is administered subcutaneously that mimics the action of hepcidin, the principal hormone regulating systemic iron homeostasis and red blood cell production.
By binding to ferroportin and inducing its degradation, rusfertide restricts iron delivery to the bone marrow, thereby reducing erythrocytosis and improving hematocrit control while markedly decreasing the need for phlebotomy.[2]
The drug has received regulatory designations including Breakthrough Therapy and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) and a New Drug Application for PV has been submitted based on these data.[3]
References
- ↑ "Rusfertide - Protagonist Therapeutics". AdisInsight. Springer Nature Switzerland AG. https://adisinsight.springer.com/drugs/800049673.
- ↑ "Modulators of the hepcidin pathway in polycythemia vera and myelofibrosis". Blood 147 (12): 1278–1288. March 2026. doi:10.1182/blood.2025028643. PMID 41100735.
- ↑ "Protagonist and Takeda Announce ASCO Plenary Presentation Highlighting Full 32-Week Results from Phase 3 VERIFY Study of Rusfertide, Showing Reductions in Phlebotomy, Improved Hematocrit Control in Polycythemia Vera". Takeda. https://www.takeda.com/newsroom/newsreleases/2025/polycythemia-vera-rusfertide-study/.
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