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Getting the latest healthcare news for you
Getting the latest healthcare news for you
The FDA has greenlit rusfertide (Mimrylo), the first drug of its kind for adults with polycythemia vera who haven't responded well to existing therapies. In the phase 3 VERIFY trial, 76.9% of patients on rusfertide needed no phlebotomy, compared to just 32.9% on placebo. This marks a significant step forward for a condition where uncontrolled blood counts can lead to life-threatening clots.
The FDA has approved rusfertide (Mimrylo), a first-in-class therapy for adults with polycythemia vera (PV) — a rare blood disorder marked by excess red blood cell production — who haven't achieved adequate control with existing treatments. Developed by Protagonist Therapeutics and Takeda, Mimrylo targets erythrocytosis and is administered as a weekly subcutaneous injection.
The approval was based on results from the randomized, phase 3 VERIFY trial, which enrolled 293 adults with PV requiring frequent phlebotomies despite standard-of-care therapy. Patients started at 19 mg weekly and were titrated to keep hematocrit below 45%. The results were compelling: rusfertide dramatically outperformed placebo in reducing the need for blood draws.
By the Numbers:
Why it matters: Uncontrolled hematocrit in PV raises the risk of serious thrombotic events. Frequent phlebotomies — the current standard — are burdensome for patients. Mimrylo offers a new, targeted option that could meaningfully reduce that burden and improve long-term disease management.
