The US Food and Drug Administration (FDA) has approved Mimrylo (rusfertide) for the treatment of adults with polycythemia vera (PV).
PV is a condition characterized by an increased production of red blood cells. Affected people may also have excess white blood cells and platelets. The excess cell levels lead to thicker blood and increased risk for serious thrombotic events and stroke. PV occurs more frequently in men than it does in women. The condition has been associated with genetic changes in the JAK2 and TET2 genes.
Rusfertide is a subcutaneous first-in-class hepcidin mimetic peptide. Its approval is based on results from the phase 3 randomized, double-blind, placebo-controlled VERIFY clinical trial (NCT05210790).
Patients treated with rusfertide achieved statistically significant benefit over those in the placebo arm, including in the primary outcome of proportion of responders achieving a durable clinical response, defined as absence of phlebotomy eligibility. Additionally, rusfertide met all four secondary endpoints; mean number of phlebotomies, proportion of participants maintaining hematocrit less than 45%, and mean change from baseline total fatigue score based on PROMIS Short Form 8a at week 32, and mean change from baseline in MFSAF total score.
The safety profile was favorable with the most common adverse reactions being injection site reactions and anemia.
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To learn more about PV and other rare hematologic disorders, visit https://checkrare.com/diseases/hematologic-disorders/