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FDA approval · Takeda · Hematology

Mimrylorusfertide

FDA approves the first hepcidin mimetic for adults with polycythemia vera.

CompareFDA press release
FDA approval · New moleculeFirst hepcidin mimetic35-second read

Mimrylorusfertide

Takeda · Hepcidin mimetic · Hematology

Bottom line for your patients

Adults with polycythemia vera who still need frequent phlebotomies despite existing therapy have a weekly injection that reduced the need for phlebotomy in its pivotal trial.

Drug brief
Drug IQ
Who it is for
Adults with polycythemia vera.
What changes
FDA describes it as a new approach for patients not adequately controlled with existing therapies, which often include phlebotomy. The pivotal trial compared it with placebo on top of ongoing standard-of-care therapy.
The evidence
Overall, 76.9% of patients on Mimrylo required no phlebotomies during the 32-week period compared to 32.9% on placebo. Efficacy was measured by the proportion who did not meet criteria for phlebotomy between weeks 20 and 32.Placebo-controlled randomized trialVERIFY · Phase 3 · 293 participantsRandomized against placebo: shows that it works, not how it compares with existing treatments.
Watch for
The most common adverse reactions were injection site reactions and anemia. The pivotal comparison ran for 32 weeks.
How it is given
Subcutaneous injectionOnce weekly. In the pivotal trial it was started at 19 mg and titrated to keep hematocrit below 45%.
What the approval tells you
New molecule. An active ingredient the FDA had not approved before.Priority review. The FDA aimed to decide in about 6 months instead of the standard 10. A faster clock, not a higher or lower evidence bar.

Clinical summary

Mimrylo is a once-weekly subcutaneous hepcidin mimetic for adults with polycythemia vera. In the phase 3 VERIFY trial of 293 adults who required frequent phlebotomies despite standard-of-care therapy, 76.9% of patients on Mimrylo required no phlebotomies compared to 32.9% on placebo. The most common adverse reactions were injection site reactions and anemia.

From the source 10

FDA press release

The FDA press release's own words, quoted exactly. Each line is what a claim on this page was checked against.

  1. The U.S. Food and Drug Administration today approved Mimrylo (rusfertide), a new treatment for adults with polycythemia vera, a rare blood disorder that causes the body to make too many red blood cells.

    Backs · Approval, adults with polycythemia vera

  2. It is the first approved treatment for polycythemia vera that mimics hepcidin, a hormone that naturally regulates iron in the body.

    Backs · First hepcidin mimetic for polycythemia vera

  3. The safety and efficacy of Mimrylo were evaluated in the VERIFY trial, a multicenter, randomized, double-blind, placebo-controlled phase 3 study of 293 adults with polycythemia vera who required frequent phlebotomies despite ongoing standard-of-care therapy.

    Backs · VERIFY, phase 3, 293 adults, placebo-controlled

View all 10 lines

  1. Patients were randomized 1:1 to receive either Mimrylo or placebo over 32 weeks.

    Backs · 32 weeks

  2. Mimrylo treatment started at 19 mg, administered subcutaneously (under the skin) once weekly, and was titrated to maintain hematocrit levels below 45%.

    Backs · 19 mg once weekly, titrated to hematocrit below 45%

  3. Efficacy was measured by the proportion of patients who did not meet criteria for phlebotomy between weeks 20 and 32 of the study.

    Backs · Endpoint window weeks 20 to 32

  4. Overall, 76.9% of patients on Mimrylo required no phlebotomies during the 32-week period compared to 32.9% on placebo.

    Backs · 76.9% vs 32.9%

  5. The most common adverse reactions were injection site reactions and anemia.

    Backs · Adverse reactions

  6. Mimrylo received priority review. The approval was granted to Takeda Pharmaceuticals America, Inc.

    Backs · Priority review

  7. Mimrylo offers a new treatment approach for patients whose disease has not been adequately controlled with existing therapies.

    Backs · Existing therapies inadequate

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