Onco360, a US independent speciality pharmacy, has been selected by Takeda Pharmaceuticals as an ultra-narrow limited distribution partner for MIMRYLO (rusfertide). The treatment is indicated for erythrocytosis in adults with polycythemia vera. MIMRYLO is a mimetic of the endogenous hormone hepcidin that blocks the iron transporter ferroportin. This inhibition reduces iron availability for red blood cell production, resulting in lower hematocrit levels. The drug's approval was based on the phase 3 VERIFY study, a global, randomised, open-label clinical trial. The primary endpoint measured the proportion of patients achieving response during weeks 20-32, defined as the absence of phlebotomy eligibility. The most common adverse reactions were injection site reactions at 56% and anaemia at 16%.

The FDA approved Takeda Pharmaceutical's MIMRYLO (rusfertide) for polycythemia vera, a blood cancer causing red blood cell overproduction. The first-in-class drug functions as a hepcidin mimetic, regulating iron distribution to control hematocrit levels and prevent life-threatening complications like strokes and pulmonary embolisms. Approval was based on the Phase 3 VERIFY study involving 293 patients. Those receiving MIMRYLO alongside standard care achieved superior hematocrit control, reduced phlebotomy needs, and improved fatigue compared to placebo. Takeda commercialises MIMRYLO under a 2024 agreement with Protagonist Therapeutics, which discovered the drug. The approval triggers $275 million in payments to Protagonist—a $200 million opt-out fee plus $75 million milestone. Protagonist remains eligible for up to $875 million in additional milestones and tiered royalties of 14% to 29% on worldwide net sales.

Takeda has received US FDA approval for MIMRYLO (rusfertide), a first-in-class treatment for erythrocytosis in adults with polycythemia vera (PV), a blood cancer. The drug is a hepcidin mimetic that regulates iron distribution and red blood cell overproduction to control hematocrit levels. The approval was supported by the Phase 3 VERIFY study involving 293 PV patients. Results showed MIMRYLO plus standard care demonstrated higher response rates than placebo, including better hematocrit control, reduced phlebotomy needs, and improved fatigue scores. PV affects approximately 90,000 people in the US. An estimated 78% of patients experience uncontrolled hematocrit with current standard care. The most common adverse events were injection site reactions and anaemia. MIMRYLO is administered once weekly via subcutaneous injection. Takeda is working with regulators outside the US for potential global availability.