Rusfertide, a first-in-class mimetic of hepcidin, shows high efficacy in the treatment of erythrocytosis polycythemia vera, with substantial improvements in hematocrit levels.
, with substantial improvements in hematocrit levels that can potentially eliminate the need for phlebotomies that are typically required – but usually ineffective.
PV, a relatively common clonal myeloproliferative neoplasm, is characterized by uncontrolled erythrocytosis, or excessive production of, increasing the risk for serious complications such as thromboembolic and cardiovascular events – the most common causes of morbidity and mortality in this blood cancer.
"This is somewhat of a paradigm shift," said Dr. Hoffman in the press briefing."We're trying to use a hormone made by the liver to control excessive red blood cell production from polycythemia vera." The second part was a withdrawal phase extending from weeks 29 to 41, in which patients were randomized in a blinded fashion to either continue on rusfertide or receive a placebo .
At the end of the second phase, 69.2% of patients receiving rusfertide were responders versus just 18.5% in the placebo group .
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