Rusfertide Is an Effective Treatment in Phlebotomy-Dependent Polycythemia Vera Patients

Rusfertide in conjunction with standard therapy is effective in managing polycythemia vera (PV) patients with poorly controlled erythrocytosis, according to a study presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting.

Researchers led by Ronald Hoffman reported results from two Phase II trials assessing the efficacy of rusfertide in PV patients. The first study consisted of 63 subjects with significant erythrocytosis despite therapeutic phlebotomy (TP) who all received rusfertide doses, 10-120 mg, were self-administered SQ week and adjusted monthly to maintain hematocrit (HCT) < 45%. The second study was comprised of 20 subjects who were given rusfertide 40 mg twice weekly and reduced to once weekly dosing when HCT < 45% was reached.

The results showed that, on rusfertide, the population of interest consistently maintained HCT < 45%, essentially eliminating TP, demonstrated normal serum ferritin, MCV values and iron deficiency. Rusfertide-treated patients also reported a statistically significant improvement in symptom burden at week 28, the researchers noted. In study 2, the average HCT was 50.7% pre-treatment and the average time to reach HCT < 45% without TP was 4.79 weeks with persistently well controlled HCT subsequently observed. Rusfertide significantly reduced erythrocyte counts following 8 weeks of treatment. The treatment was well tolerated in both studies.

“Rusfertide, when added to standard therapy, demonstrated robust activity in managing PV patients with sub-optimally controlled erythrocytosis in 2 trials,” the researchers concluded. “Taken together, these data show that the non-cytoreductive rusfertide, is a promising novel agent for PV patients which leads to sustained HCT control < 45%.”