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

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 IDH1^R132 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