FDA Approves First-in-Class Drug for Polycythemia Vera

THURSDAY, Sept. 3, 2026 (HealthDay News) — The U.S. Food and Drug Administration has approved Mimrylo (rusfertide) as a new treatment for adults with polycythemia vera who have not achieved adequate control with existing therapies.

Mimrylo mimics hepcidin, a hormone that naturally regulates iron in the body, and limits the iron available to make new red blood cells, thereby reducing red blood cell overproduction.

The approval was based on results from the phase 3 VERIFY trial that included 293 adults with polycythemia vera who required frequent phlebotomies despite ongoing standard-of-care therapy. Patients were randomly assigned to receive either Mimrylo (started at 19 mg, administered subcutaneously once weekly, and titrated to maintain hematocrit <45 percent) or placebo. From 20 to 32 weeks, 76.9 percent of patients on Mimrylo required no phlebotomies versus 32.9 percent on placebo. The most common adverse reactions included injection site reactions and anemia.

“People living with polycythemia vera have long faced the challenge of managing a chronic blood disorder with frequent blood draws to help address the consequences of the red blood cell overproduction,” Tanya Wroblewski, M.D., from the FDA Center for Drug Evaluation and Research, said in a statement. “Today”s approval of Mimrylo offers a new, first-in-class option that has the potential to meaningfully reduce patient burden.”

Approval of Mimrylo was granted to Takeda Pharmaceuticals.

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