Rusfertide Poised to Transform Polycythemia Vera Care: Andrew Kuykendall, MD

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Andrew T. Kuykendall, MD, lead investigator for the phase 3 VERIFY trial (NCT05210790), emphasizes the critical need for therapies that offer a sense of normalcy to individuals with polycythemia vera, elaborating on the unique challenges of living with polycythemia vera and examining the profound impact of rusfertide on these patients.

Early indications from the phase 2 REVIVE trial (NCT04057040), findings which served as a foundation for VERIFY, were overwhelmingly positive, with patients reporting they felt "better than they ever had; this was really something that changed their lives," Kuykendall explains. This strong, meaningful feedback centered on improvements to their symptoms and their daily lives, allowing them to regain "a sense of independence and freedom that they didn't have before or they thought they'd lost,” he adds.

Beyond the goal of curing polycythemia vera and targeting its underlying disease-driving mutations, there's a growing recognition of the need to address what matters to patients on a daily basis. This agent and trial, Kuykendall notes, were unique in their focus on clinically relevant end points, aiming to determine if a better approach was possible. Core questions driving the research were: can blood counts be controlled? Can patients be freed up from the health care system? Is it possible to move away from the need for phlebotomies? Is it possible to make patients feel better on a daily basis? Phlebotomy, a deeply entrenched treatment paradigm, is common for patients often managed in the community, Kuykendall explains.

These objectives resonated deeply with patients and are precisely why the results, announced at the American Society of Clinical Oncology annual meeting, were deemed so exciting. The potential for rusfertide’s self-injection approach to really alter community physicians’ practice on a daily basis with some of these patients, he concludes, makes it a topic of great interest.

Rusfertide most recently received a breakthrough therapy designation from the FDA on August 25.¹ It previously received an orphan drug designation and a fast track designation in 2020.²

Revisit part 1 of this interview, in which Kukendall speaks to rusfertide’s potential as a sustainable management strategy for polycythemia vera, and part 2, in which he first alludes to the need for therapies to treat this myeloproliferative neoplasm to offer a sense of normalcy.

References

1. Pelosci A. Rusfertide earns FDA BTD for erythrocytosis in polycythemia vera. Cancer Network^®. August 25, 2025. Accessed September 2, 2025. https://www.cancernetwork.com/view/rusfertide-earns-fda-btd-for-erythrocytosis-in-polycythemia-vera
2. Rusfertide received breakthrough therapy designation for treatment of erythrocytosis in patients with polycythemia vera. The National Law Review. August 25, 2025. Accessed September 2, 2025. https://natlawreview.com/press-releases/rusfertide-receives-breakthrough-therapy-designation-treatment