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 U.S. Food and Drug Administration (FDA) accepted a supplemental new drug application and granted priority review to oral ivosidenib tablets, a potent and targeted inhibitor of mutant IDH1, for the treatment of patients with IDH1-mutated relapsed/refractory (R/R) myelodysplastic syndromes (MDS).¹

Oral ivosidenib has previously been approved by the FDA as a monotherapy and in combination with azacitidine for the treatment of patients aged ≥75 years or who are ineligible for intensive induction chemotherapy with newly diagnosed (ND) IDH1-mutated acute myeloid leukemia (AML).¹ Oral ivosidenib was also recently approved by the European Commission for a subset of the same patient population: those with IDH1^R132 mutations.¹

This supplemental new drug application is based on results from a multicenter, open-label, phase I study (NCT02074839), previously covered by the MDS Hub, that found oral ivosidenib produced durable remissions and an acceptable safety profile in patients with R/R IDH1-mutated MDS.¹