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What is ivosidenib?
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• June 26, 2023
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What is Ivositinib? Ivositinib is a medication called an IDH1 inhibitor. This is an example in oncology of targeted therapy. So not every patient is eligible to respond to Ivositinib, and only patients who have a mutation in a gene called IDH1 are eligible and would respond to Ivositinib. What is an IDH1 mutation? IDH1 is a mutation that is found in about 5 to 10 percent of patients who have acute myeloid leukemia. It causes a problem in some of the metabolism of different blood cells that creates this product called 2-Hg, which makes it so that cells can grow out of control and lead to leukemia. IDH1 is found not in every patient, but when it is found, it is an indication that this is a patient who may be benefiting from treatment with an IDH1 inhibitor. How would a patient know if they have this mutation? A patient would know that they have an IDH1 mutation based on different mutational tests that we do. This is frequently done at diagnosis at the time of a bone marrow biopsy, where we send a test called next-generation sequencing to understand what sort of gene mutations their leukemia harbors. There's other ways to detect these mutations throughout the course of someone's disease, but it is usually done from either a blood test or a bone marrow test. When during the course of treatment would someone with an IDH1 mutation receive Ivositinib? So Ivositinib can be given both in the first part of treatment, called induction, or if leukemia relapses. And IDH1 can be given in both of these settings and has shown to be beneficial and have activity and can treat both initially newly diagnosed leukemia or AML that has relapsed after previous treatments. How is Ivositinib administered? Ivositinib is a pill that is taken every day, and it is a pill that is taken just with water and can be very well tolerated, but it is often combined with other medications to improve the effectiveness of Ivositinib. What is it usually used in combination with? So Ivositinib is effective by itself, just given by itself either in newly diagnosed patients or in patients who have relapsed. But we've also learned that Ivositinib is even more effective if given with other medications. And mostly these are medications called hypomethylating agents, specifically a medication called azacytidine, which is an injection or infusion that's given. And the combination of both of those drugs really makes it so that Ivositinib works better when given with this medication.

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