US FDA approves Takeda, Protagonist’s drug for rare blood cancer

By Kamal Choudhury and Mariam Sunny

Aug 28 (Reuters) – The U.S. Food and Drug Administration has approved a first-of-its-kind drug from Takeda Pharmaceutical and Protagonist Therapeutics to treat ​a rare blood cancer, the health regulator said on ‌Friday.

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.

(Reporting by Kamal Choudhury, Mariam Sunny and Christy Santhosh in Bengaluru; Editing ​by Vijay Kishore)