FDA approves first-in-class drug for polycythemia vera
The U.S. Food and Drug Administration has approved Takeda Pharmaceuticals' Mimrylo (rusfertide) as a new treatment for adults with polycythemia vera who have not achieved adequate control with existing therapies.
The U.S. Food and Drug Administration has approved a groundbreaking drug, Takeda Pharmaceuticals' Mimrylo, for adults with polycythemia vera who have not seen improvement with current treatments. Mimrylo, also known as rusfertide, functions by mimicking hepcidin, a hormone that naturally controls iron levels in the body, effectively limiting the iron necessary for the creation of new red blood cells. This results in a decrease in red blood cell overproduction.
The approval was supported by the findings from the Phase III VERIFY trial, which involved 293 adults with polycythemia vera needing regular phlebotomies even with ongoing standard care. Participants were randomly assigned to receive either Mimrylo (starting at 19 mg, administered subcutaneously weekly and adjusted to maintain hematocrit below 45%) or a placebo. From weeks 20 to 32, 76.9% of Mimrylo recipients required no phlebotomies, compared to 32.9% on placebo.
The most frequently reported side effects were injection site reactions and anemia. Tanya Wroblewski, M.D., from the FDA Center for Drug Evaluation and Research, expressed that Mimrylo offers a new, first-of-its-kind treatment that could significantly reduce the burden on patients with this chronic blood disorder.
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