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

Weekly subcutaneous injection with rusfertide, a hepcidin mimetic, significantly reduced the need for phlebotomy and improved quality-of-life in patients 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 hematocrit (Hct) below 45% [1], typically through therapeutic phlebotomy and cytoreductive therapy. However, frequent phlebotomy can be burdensome and often impairs quality-of-life.

Rusfertide mimics the action of hepcidin, the master regulator of iron homeostasis. In the earlier phase 2 REVIVE trial, rusfertide demonstrated efficacy in controlling Hct and reducing phlebotomy requirements [2]. The randomised, placebo-controlled phase 3 VERIFY study (NCT05210790) enrolled 293 patients with PV and assigned them in a 1:1 ratio to receive the current standard-of-care plus weekly subcutaneous rusfertide or placebo [3]. The primary endpoint was phlebotomy independence during weeks 20–32. Dr Andrew Kuykendall (Moffitt Cancer Center, FL, USA) presented the initial results [3].

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 target Hct <45% through week 32 than those on placebo (62.6% vs 14.4%; P<0.0001). Symptom burden, measured by the Myelofibrosis Symptom Assessment Form (MFSAF TSS7), also improved more with rusfertide (mean change: -2.40) than with placebo (mean change: -0.54; P=0.0239).

“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). LBA3, ASCO Annual Meeting 2025, 30 May–3 June, Chicago, IL, USA.

Medical writing support was provided by Dr Marten Dooper.
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Posted on 4 June 20254 June 2025