Takeda and Protagonist Therapeutics have submitted a New Drug Application (NDA) to the FDA seeking approval of rusfertide for the treatment of adults with polycythemia vera (PV), a chronic blood cancer marked by overproduction of red blood cells and increased thrombotic risk. Rusfertide is a first-in-class investigational hepcidin mimetic given as a once-weekly subcutaneous injection and is designed to regulate iron homeostasis and red blood cell production to help control hematocrit levels.
The NDA is backed by 52-week data from the Phase 3 VERIFY study, a global randomized, placebo-controlled trial in 293 patients with PV who were phlebotomy-dependent despite standard therapies. In VERIFY, rusfertide plus standard of care met the primary endpoint and all four key secondary endpoints, including durable hematocrit control, reduced need for therapeutic phlebotomy, and improvements in pre-specified patient-reported outcomes. The application also incorporates findings from the Phase 2 REVIVE study and ongoing long-term follow-up in the THRIVE extension trial.
Rusfertide has previously received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for PV. Under a 2024 global license and collaboration agreement, Protagonist and Takeda are co-developing and plan to co-commercialize rusfertide if approved.
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