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On October 24, 2023, the U.S. Food and Drug Administration (FDA) approved ivosidenib, an isocitrate dehydrogenase-1 (IDH1) inhibitor, for the treatment of adult patients with relapsed/refractory myelodysplastic syndromes with an IDH1 mutation detected by an FDA-approved test.1 The FDA also approved a RealTime IDH1 Assay as a companion diagnostic.1
Ivosidenib was previously granted priority review by the FDA for the same indication, and it has previously been approved by the FDA as a monotherapy and in combination with azacitidine. This approval is for patients either aged ≥75 years or ineligible for intensive chemotherapy with newly diagnosed IDH1-mutated acute myeloid leukemia. Ivosidenib was also previously approved by the European Commission for a specific subgroup within this patient population: those with IDH1R132 mutations.
This approval is based on results from an open-label, multicenter phase I trial (NCT02074839) which was previously covered by the MDS Hub.1 The complete/partial remission rate was 39% (n = 18), with a median duration of complete remission ranging from 1.9 to 80.8 months.1 Treatment-related adverse events were similar for patients with myelodysplastic syndromes and those with acute myeloid leukemia treated with ivosidenib monotherapy.1 Ivosidenib comes with a boxed warning for differentiation syndrome.1
FDA grants priority review and accepts supplemental new drug application for oral ivosidenib for the treatment of patients with IDH1-mutated R/R MDS
On August 15, 2023, the FDA accepted a supplemental new drug application and granted priority review...
Updates from #ASCO23 and EHA on the efficacy and safety of ivosidenib, including molecular characterization and long-term follow-up in patients with mutant IDH1 AML and R/R MDS
At the ASCO and EHA congresses, updates were presented on the efficacy...
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