FDA approves rusfertide for rare blood cancer polycythemia vera

The U.S. Food and Drug Administration has approved rusfertide, branded as Mimrylo, for the treatment of adults with polycythemia vera. Developed by Takeda Pharmaceutical and Protagonist Therapeutics, the drug is a first-in-class option for this rare, slow-growing blood cancer that causes the bone marrow to overproduce red blood cells.

Clinical data from the phase 3 VERIFY trial indicated that 76.9% of patients receiving rusfertide did not require phlebotomies (bloodletting) during a 32-week period, compared to 32.9% of those receiving a placebo. The treatment aims to reduce the patient burden associated with frequent blood draws used to manage the risks of blood clots, strokes, and heart attacks.

Takeda expects peak global sales for the drug to reach between $1 billion and $2 billion. While the exact list price has not been disclosed, company officials stated the pricing would aim to balance value with patient access.