Updated NCCN guidelines now recommend rusfertide as a Category 1 option for both low- and high-risk polycythemia vera.
Rusfertide (Mimrylo) has been recommended as a Category 1 treatment option across both low- and high-risk polycythemia vera in the latest National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for Myeloproliferative Neoplasms.1
The recommendation makes rusfertide the only therapy to carry a Category 1 designation across both high- and low-risk polycythemia vera categories. The inclusion is based on findings from both the phase 3 VERIFY trial (NCT05210790) and the phase 2 REVIVE trial (NCT04057040).
In part 1a of VERIFY, patients who received rusfertide plus current standard of care (n = 147) achieved a clinical response in 76.9% of cases compared with 32.9% among those given placebo plus standard of care (n = 146) during weeks 20 to 32 (P < .0001).2
“The inclusion of rusfertide in the NCCN Guidelines is a meaningful milestone for patients with polycythemia vera, who have long needed more options,” said Rushi Potdar, MD, chief medical officer and head of global medical affairs of Takeda Oncology in a news release.1 “Rusfertide’s availability in the U.S. marks a potential shift in the treatment paradigm for polycythemia vera, giving clinicians and patients access to a novel therapy with the potential to help maintain hematocrit control, which the majority of patients being treated for polycythemia vera do not achieve today.”
Notably,
VERIFY is an ongoing, 3-part, global, randomized, trial that included patients who were 18 years of age with polycythemia vera who had uncontrolled hematocrit and were phlebotomy-dependent.4
In Part 1a of the trial, patients were randomly assigned 1:1 to receive once-weekly, self-administered subcutaneous rusfertide or placebo, in combination with their current standard of care from weeks 20 to 32.5 For part 1b in weeks 32 to 52, all patients received rusfertide plus standard of care, with part 2 being a open-label, long-term safety evaluation period.
The primary end point was the proportion of patients achieving a response during weeks 20 to 32, defined as the absence of phlebotomy eligibility.1 Key secondary end points, assessed at week 32, included the mean number of phlebotomies, patients maintaining hematocrit below 45%, and mean changes in fatigue. Importantly, all patients have completed the randomized portion of VERIFY and are now in the open-label extension.
REVIVE, evaluated the rusfertide in patients who were at least 18 years old with phlebotomy-requiring polycythemia vera.6 Patients also needed to have received at at least 3 phlebotomies for inadequate hematocrit control in the 6 months prior to randomization or 5 in the year prior.
The trial evaluated rusfertide across 3 parts, the first of which included a 28-week, open-label, dose-finding part 1 (n = 70), then a blinded, placebo-controlled, randomized withdrawal part 2 (n = 59), and finally a 52-week, open-label expansion part 3 (n = 58).1
Rusfertide met both the primary end point and all secondary end points in the VERIFY trial.4
Rusfertide was generally well tolerated through 52 weeks of treatment in the VERIFY trial.1 The most common adverse effects occurring in more than 15% of patients were injection site reactions (56%) and anemia (16%). In VERIFY specifically, injection site reactions occurred in 47.4% of patients and anemia occurred in 25.6% of patients.4
