WASHINGTON, D.C. — The Food and Drug Administration approved Takeda Pharmaceuticals America Inc.’s Mimrylo for adults with polycythemia vera whose disease is not adequately controlled with existing therapy, introducing a first-in-class treatment designed to reduce red blood cell production and lessen reliance on repeated phlebotomy.
Mimrylo, or rusfertide, is the first approved therapy for polycythemia vera that mimics hepcidin, a hormone that regulates iron availability. By restricting iron used to produce new red blood cells, the drug is intended to keep hematocrit levels below the threshold associated with increased cardiovascular risk.
Polycythemia vera is a rare blood disorder in which the body produces too many red blood cells, thickening the blood and increasing the risk of complications including blood clots, stroke and heart attack.
Treatment commonly includes phlebotomy, in which blood is removed to lower red blood cell levels. Some patients continue to require frequent procedures despite standard therapy.
“Today’s approval of Mimrylo offers a new, first-in-class option that has the potential to meaningfully reduce patient burden,” Tanya Wroblewski, director of the FDA’s Division of Nonmalignant Hematology, stated.
The FDA based its decision on the Phase 3 VERIFY trial, which enrolled 293 adults with polycythemia vera who continued to require frequent phlebotomies despite standard-of-care treatment.
Patients were randomly assigned to Mimrylo or placebo for 32 weeks. Mimrylo was administered by a weekly subcutaneous injection beginning at 19 milligrams, with dosing adjusted to maintain hematocrit below 45%.
The study measured whether patients avoided meeting criteria for phlebotomy between weeks 20 and 32.
Overall, 76.9% of patients receiving Mimrylo required no phlebotomies during the 32-week study period, compared with 32.9% of patients receiving placebo, according to the FDA.
The most frequently reported adverse reactions were injection-site reactions and anemia.
The FDA granted Mimrylo Priority Review before approving the treatment.
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