FDA accepted NDA and granted priority review for rusfertide in PV

On March 2, 2026, the U.S. Food and Drug Administration (FDA) accepted the new drug application (NDA) and granted priority review for rusfertide, a hepcidin mimetic peptide, for the treatment of adult patients with polycythemia vera (PV).¹ The NDA acceptance was based on results from the phase III VERIFY study (NCT05210790), the phase II REVIVE study (NCT04057040), and the long-term extension THRIVE study (NCT06033586).¹  

Topline results of the VERIFY study have previously been reported by the MPN Hub, in which rusfertide demonstrated a greater clinical response rate vs placebo (76.9% vs 32.9%; p < 0.0001).² Serious adverse events (SAEs) occurred in 3.4% of patients in the rusfertide group vs 4.8% of patients in the placebo group.² Final results from the REVIVE study have also previously been covered by the MPN Hub; rusfertide demonstrated greater hematocrit control without therapeutic phlebotomy vs placebo (69.2% vs 14.8%; p < 0.0001).³ Grade 3 treatment emergent adverse events (TEAEs) occurred in 25.7% of patients in the REVIVE study.³ Results from THRIVE, a long-term extension of REVIVE, have also been reported by the MPN Hub. The median treatment duration across REVIVE and THRIVE was 196 weeks, with mean hematocrit remaining stable at 42.3% (standard error of the mean [SEM], 0.54) at the end of REVIVE and 43.0% (SEM, 1.34) at Week 60 of THRIVE.⁴ The most common adverse events (AEs) were Grade 1–2 injection site reactions (87%), fatigue (61%), and COVID‑19 (52%).⁴