FDA Approval Summary: Ivosidenib for Treatment of Adult Patients with Relapsed/Refractory Myelodysplastic Syndrome with an IDH1 Mutation.

Abstract

On October 24, 2023, the FDA approved a supplemental application for ivosidenib (Tibsovo; Servier) for the treatment of adult patients with relapsed/refractory (R/R) isocitrate dehydrogenase 1-mutated myelodysplastic syndrome (MDS). The efficacy of ivosidenib in patients with R/R MDS was established in study AG120-C-001, an open-label, single-arm trial, based on the complete remission (CR) + partial remission (PR) rate, duration of CR + PR, and conversion of transfusion dependence to transfusion independence. With a median follow-up of 27.1 months in 18 patients with isocitrate dehydrogenase 1-mutated R/R MDS treated with ivosidenib 500 mg once daily, the CR + PR rate was 39% (95% confidence interval, 17.3-64.3; all CR responses), with a median duration of response not estimable (range, 1.9-80.8+ months). Of the nine patients with transfusion dependence at baseline, six achieved transfusion independence (67%). Serious adverse reactions in ≥5% included differentiation syndrome, fatigue, and rash. The most common (≥20%) adverse reactions were arthralgia, fatigue, cough, diarrhea, decreased appetite, mucositis, myalgia, and pruritus.