FDA approves Takeda and Protagonist's rusfertide for rare blood cancer, ending decades of reliance on bloodletting

The US Food and Drug Administration has approved rusfertide, a first-of-its-kind treatment for polycythemia vera developed by Takeda Pharmaceutical (TAK) and Protagonist Therapeutics (PTGX), offering thousands of patients a less invasive alternative to the frequent blood removal procedures that have long defined management of the condition.

Branded as Mimrylo, the drug treats a rare, slow-growing blood cancer that affects roughly 90,000 Americans. The disease causes the bone marrow to overproduce red blood cells, thickening the blood and sharply raising the risk of life-threatening clots, strokes, and heart attacks. For decades, physicians have relied primarily on therapeutic phlebotomy — the regular removal of blood — to keep red blood cell levels in check.

Mimrylo works by mimicking hepcidin, the body's master iron-regulating hormone, restricting iron availability and curbing excess red blood cell production. In late-stage trials, the drug, administered alongside standard of care, helped patients control red blood cell levels, reduced the need for blood draws, and improved symptoms including fatigue.

Teresa Bitetti, president of Takeda's global oncology business unit, said the drug would be made available to patients within 48 hours of approval. It is priced at approximately $4,200 per vial. Bitetti said the company sought to price the treatment in a manner that reflected its clinical value while preserving patient access.

Takeda projects peak global sales of between $1bn and $2bn for the drug, which it secured rights to co-develop and commercialize through a licensing agreement with Protagonist signed in 2024.