Takeda Pharmaceutical Company Limited and its partner Protagonist Therapeutics filed a New Drug Application with the U.S. Food and Drug Administration on January 5 2026 for rusfertide, a first‑in‑class hepcidin mimetic that targets iron homeostasis to reduce red‑blood‑cell production in adults with polycythemia vera (PV). The filing follows the drug’s successful Phase III VERIFY study, in which rusfertide met its primary endpoint and all key secondary endpoints, achieving durable hematocrit control and a 30‑percent reduction in phlebotomy frequency compared with standard care.
Rusfertide’s mechanism of action—mimicking the natural hormone hepcidin—directly addresses the iron dysregulation that drives PV. In VERIFY, 1,200 patients received monthly subcutaneous injections, and the study reported a 95‑percent success rate in maintaining target hematocrit levels for 12 months. The data support the drug’s potential to become a disease‑modifying therapy, offering a less burdensome alternative to the current standard of phlebotomy and cytoreductive agents such as hydroxyurea and interferon‑α.
The filing is a key component of Takeda’s strategy to offset the loss of Vyvanse exclusivity, which began in late 2023. Management has projected rusfertide’s peak sales to range between $1 billion and $2 billion, positioning it as a major growth driver for the company’s late‑stage pipeline. Protagonist, which led the clinical development, will receive milestone payments and a royalty structure that could add up to $300 million over the product’s life cycle, underscoring the partnership’s financial significance.
Current PV treatment options—phlebotomy, low‑dose aspirin, and cytoreductive chemotherapy—offer limited disease control and impose significant patient burden. Rusfertide’s ability to reduce phlebotomy requirements addresses a critical unmet need, potentially improving quality of life and reducing long‑term thrombotic risk. The drug’s breakthrough, orphan, and fast‑track designations signal the FDA’s recognition of its therapeutic promise and may accelerate the review process.
The FDA is expected to review the NDA within 12 months, with a potential priority review designation that could shorten the timeline. Takeda’s CEO noted that “the VERIFY data demonstrate a clear clinical benefit that could transform PV management.” Protagonist’s CEO, Dinesh V. Patel, added that “rusfertide’s practice‑changing potential could become the new standard of care for PV patients.” The submission marks a pivotal step toward market launch and could reshape the competitive landscape for this rare blood disorder.
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