Rusfertide improved symptoms and quality-of-life in polycythaemia vera

Weekly subcutaneous injection with rusfertide, a hepcidin mimetic, significantly reduced the need for phlebotomy and improved the quality-of-life of participants with phlebotomy-dependent polycythaemia vera (PV), according to results from the phase 3 VERIFY trial.

PV is a rare haematologic disease characterised by low hepcidin levels, systemic iron deficiency, and excessive erythrocyte production, leading to elevated thrombotic risk. The primary goal of treatment is to reduce this risk by maintaining haematocrit (Hct) levels below 45%, typically through therapeutic phlebotomy and cytoreductive therapy [1]. However, frequent phlebotomy can be burdensome and often impacts patients’ quality-of-life. Rusfertide mimics the action of hepcidin, the master regulator of iron homeostasis.

In the earlier phase 2 REVIVE trial (NCT04057040), rusfertide demonstrated efficacy in controlling Hct and reducing phlebotomy requirements [2]. The randomised, placebo-controlled phase 3 VERIFY study (NCT05210790) followed up on that, and Dr Andrew Kuykendall (Moffitt Cancer Center, FL, USA) presented the initial results [3].

VERIFY enrolled 293 participants with PV and randomly assigned them 1:1 to receive the current standard-of-care plus weekly subcutaneous rusfertide or placebo. The primary endpoint was phlebotomy independence during weeks 20–32.

Among participants receiving rusfertide, 113 (76.9%) remained phlebotomy-free between weeks 20 and 32, compared with 48 (32.9%) in the placebo group (P<0.0001). Moreover, significantly more participants in the rusfertide arm achieved a target Hct <45% by week 32 than those on placebo (62.6% vs 14.4%; P<0.0001; see Figure). Symptom burden, measured by the Myelofibrosis Symptom Assessment Form TSS7, was more pronouncedly reduced with rusfertide (mean change -2.40) compared with placebo (mean change -0.54; P=0.0239).

Figure: Rusfertide combined with standard-of-care is more likely to maintain haematocrit levels <45% from weeks 0–32 than the placebo control in participants with polycythaemia vera [3]



BL, baseline; CSC, current standard-of-care; Hct, haematocrit; PBO, placebo; SEM, standard error of measurement.

“These findings suggest that rusfertide could represent a promising new treatment option for patients with PV,” Dr Kuykendall concluded.

1. Tremblay D, et al. JAMA. 2025;333(2):153–160.
2. Kremyanskaya M, et al. N Engl J Med 2024;390(8);723–735.
3. Kuykendall AT, et al. Results from VERIFY, a phase 3, double-blind, placebo (PBO)-controlled study of rusfertide for treatment of polycythemia vera (PV). Abstract LBA3, ASCO Annual Meeting 2025, 30 May–3 June, Chicago, IL, USA.

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Posted on 30 July 2025