On August 28, Takeda announced that Rusfertide (trade name: Mimrylo) has been approved by the FDA for the treatment of adult patients with polycythemia vera (PV).
Rusfertide is a first-in-class hepcidin analogue , acquired by Takeda from Protector Therapeutics . It is designed to regulate iron distribution and excessive erythrocyte production in the body to control hematocrit (the ratio of red blood cells to total blood volume). The primary treatment goal for PV is to keep hematocrit below 45%. Takeda holds the exclusive global rights to develop and commercialize Rusfertide .
This approval is supported by data from the global Phase III VEVERIFY study. This study was a randomized, multicenter, placebo-controlled clinical trial (n=293) designed to evaluate the efficacy and safety of rusfertide (once weekly, subcutaneously) in patients with PV. The primary endpoint of the study was response rate (the proportion of patients who did not require phlebotomy).
The results showed that the study met all efficacy endpoints. During weeks 20-32 of treatment, the response rates in the Rusfertide group and the placebo group were 77% and 33%, respectively. During weeks 0-32 of treatment, the mean number of phlebotomies was 0.5 per patient in the Rusfertide group and 1.8 per patient in the placebo group. Furthermore, Rusfertide demonstrated a good safety profile, with the most common treatment-emergent adverse events (TEAEs) being injection site reactions and anemia.
Approximately 90,000 people in the United States are affected by polycythemia vera (PV), characterized by excessive red blood cell production (erythrocytosis), leading to an elevated hematocrit, which increases blood viscosity or thickness. This can result in life-threatening thrombotic events, including stroke, deep vein thrombosis, and pulmonary embolism. Maintaining hematocrit levels below 45% can prevent thrombotic events and alleviate severe symptoms, including severe fatigue, itching, difficulty concentrating, and night sweats. It is estimated that even with current standard treatments (including phlebotomy and erythropoiesis), 78% of patients still experience uncontrolled hematocrit . PV patients with uncontrolled hematocrit have a four times higher risk of cardiovascular death or major cardiovascular events.

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