USFDA Approves Takeda, Protagonist's Mimrylo for Rare Blood Cancer Polycythemia Vera

The approval of rusfertide brings a ‌highly anticipated, less-invasive treatment ⁠option ⁠for thousands of patients who have historically relied on frequent bloodletting procedures to manage their condition.

The drug treats polycythemia vera, a rare, slow-growing blood cancer affecting roughly 90,000 ​Americans. The condition 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.

Rusfertide, branded as Mimrylo, will be ⁠made available ‌to patients within 48 hours of FDA approval, Teresa Bitetti, president of Takeda's global oncology business unit, told Reuters in an ⁠interview.

While the company did not disclose the exact list ​price of the drug at launch, Bitetti said the ​company would "price this in a way that is fair to the value, but then also ensures that there's access for patients."

Takeda is projecting peak global sales for the drug in the range of $1 billion to $2 billion.

For decades, treatment has largely relied on therapeutic phlebotomy, a procedure ‌that involves regularly removing blood to keep red blood cell levels under control.

Mimrylo works by mimicking hepcidin, the ​body's master iron-regulating ​hormone, to restrict iron ⁠availability and prevent the bone marrow from overproducing red blood cells.

The FDA's decision was based on late-stage trials, in which the drug, alongside standard ​of care, helped patients control their red blood cell levels, reducing the need for painful blood draws, and improving symptoms like fatigue.

Takeda secured the global rights to co-develop and commercialize the drug from its developer Protagonist through a 2024 licensing agreement.