FDA Approves Rusfertide in Polycythemia Vera

Rusfertise (Mimrylo) has been approved by the FDA for adults patients with polycythemia vera, according to a press release from the FDA.¹

Results from the phase 3 VERIFY trial (NCT05210790) assessing 293 patients with polycythemia vera led to the approval.² Of note, 76.9% of patients who received rusfertide did not require phelobotomies during the 32-week period compared with 32.9% of patients who received placebo. Overall, patients most commonly experienced injection site reactions and anemia.

“People living with polycythemia vera have long faced the challenge of managing a chronic blood disorder with frequent blood draws to help address the consequences of the red blood cell overproduction,” Tanya Wroblewski, MD, director of the Division of Nonmalignant Hematology within the FDA’s Center for Drug Evaluation and Research, said in the press release. “Today's approval of [rusfertide] offers a new, first-in-class option that has the potential to meaningfully reduce patient burden."

Findings from the 2025 American Society of Hematology Annual Meeting and Exposition showed that at 52 weeks, 61.9% of patients who were given rusfertide plus standard of care had an enduring clinical response. Additionally, 84.1% of patients who had a response with rusfertide between weeks 20 to 32 had a sustained response. A total of 77.9% of patients who crossed over at week 32 had a clinical response.

“Rusfertide is going to be part of our armamentarium going forward for polycythemia vera. This is an agent that is extremely well tolerated; that’s probably to be decided by the patients, but it certainly seems like that,” Andrew Kuykendall, MD, an assistant member in the Department of Malignant Hematology at Moffitt Cancer Center and lead investigator of the VERIFY trial, stated in an interview with CancerNetwork® ahead of the approval. “This is something that could be a part of every patient with polycythemia vera’s treatment history, maybe not forever but at least at some point during their disease. It’s something that can be combined with other therapies that can be used for their disease too. It’s something that we’re going to be talking about a lot with patients.”

References

1. FDA approves first drug of its kind for polycythemia vera, a rare blood disorder. News release. FDA. August 28, 2026. Accessed August 28, 2026. https://tinyurl.com/yeavrkc4
2. Protagonist and Takeda present longer-term data at ASH 2025 showing rusfertide delivers durable response and hematocrit control in polycythemia vera. News release. Takeda. December 6, 2025. Accessed August 28, 2026. https://tinyurl.com/5n9ahj8r