FDA approves Mimrylo, first hepcidin-mimicking drug for polycythemia vera

The U.S. Food and Drug Administration announced approval of Mimrylo (rusfertide) for adult patients with polycythemia vera, a rare blood disorder that causes excess red blood cells.

The drug mimics the hormone hepcidin, limiting iron availability and thereby reducing red blood cell production, a novel mechanism for this condition.

Mimrylo is aimed at patients whose disease is not adequately controlled with existing therapies, providing a new option to keep hematocrit levels below the target threshold.

FDA officials said the approval could lessen the need for frequent blood draws and lower the risk of complications such as blood clots, stroke, and heart attack.