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The Food and Drug Administration has granted Orphan Drug designation to inecalcitol for the treatment of patients with acute myeloid leukemia, intended to be used in combination with the hypomethylating anticancer drugs azacytidine or decitabine.

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Inecalcitol is a vitamin D receptor agonist that works with the hypomethylating agents to limit the progression of leukemia.


Information from the manufacturer, Hybrigenics, notes that a Phase II study of inecalcitrol in combination with either azacytidine or decitabine in older or frail patients with AML is currently being planned to take place both in France and the United States.


Phase II trial data for inecalcitrol used to treat patients with chronic lymphocytic leukemia has previously shown that the drug had an inhibitory effect in half of the patients; and another Phase II clinical trial to evaluate the drug used in combination with imatinib for the treatment of patients with chronic myeloid leukemia recently launched, with enrollment ongoing.