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Ivosidenib granted FDA approval for the treatment of patients with R/R IDH1-mutated MDS

Oct 25, 2023
Learning objective: After reading this article, learners will be able to cite a new development in MDS.

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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

  1. U.S. Food and Drug Administration. FDA approves new therapy for rare form of blood cancers called myelodysplastic syndromes. Published Oct 24, 2023. Accessed Oct 25, 2023.  

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