Dr Kuykendall on the Significance of the FDA Approval of Rusfertide for Polycythemia Vera

“Patients want to be outside the office and have control of their health, and rusfertide offers that… Rusfertide is a treatment that is driven by patient feedback… [The FDA approval of rusfertide] is a big win for the [polycythemia vera] community.”

Andrew Kuykendall, MD, an associate member in the Department of Malignant Hematology at Moffitt Cancer Center, discussed how the FDA approval of rusfertide (Mimrylo) for adults with polycythemia vera could provide patients with greater independence and help address burdens associated with hematocrit management.

The approval was supported by the phase 3 VERIFY trial (NCT05210790), which evaluated rusfertide in patients with polycythemia vera that required frequent phlebotomy. Results showed that 76.9% of patients who received rusfertide (n = 147) achieved a clinical response vs 32.9% in those who received placebo (n = 146; P < .0001). Additionally, hematocrit was maintained below 45% in 62.6% vs 14.4% of patients in each arm, respectively.

Kuykendall explained that patients with polycythemia vera often want more autonomy and less dependence on repeated office visits and phlebotomies. Due to rusfertide ability be self-administered by patients, he said the therapy may help patients maintain hematocrit control while spending less time with phlebotomies.

Beyond hematocrit control, Kuykendall emphasized the importance of fatigue and other disease-related symptoms. He characterized fatigue as a major contributor to impaired quality of life, noting that it can prevent patients from getting out of bed, working, or maintaining their usual schedules. Although reducing thrombotic risk remained an important clinical objective, he explained that patients may not directly feel that benefit in the same way they experience improvements in fatigue.

Reflecting on rusfertide’s development, Kuykendall noted that patients reported feeling better during a phase 2 trial, encouraging investigators to evaluate those outcomes further in a phase 3 trial. Kuykendall ultimately described rusfertide as a treatment shaped by patient feedback and characterized its approval as a win for the polycythemia vera community.