FDA Approval Summary: Ivosidenib for Treatment of Adult Patients with Relapsed/Refractory Myelodysplastic Syndrome with an IDH1 Mutation
Ashley C. Woods, E. Dianne Pulte, Xin Wang, Jonathon Vallejo, Ritu Chadda, Nan Zheng, Javier G. Blanco, Sarah E. Dorff, Hongshan Li, Jiang Liu, Olanrewaju O. Okusanya, Richard Pazdur, Marc R. Theoret, R. Angelo de Claro, Kelly J. NorsworthyAbstract
On October 24th, 2023, the FDA approved a supplemental application for ivosidenib (Tibsovo; Servier) for the treatment of adult patients with relapsed/refractory (R/R) IDH1-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 complete remission (CR) + partial remission (PR) rate, duration of CR + PR, and conversion of transfusion dependence (TD) to transfusion independence (TI). With a median follow-up of 27.1 months in 18 patients with IDH1-mutated R/R MDS treated with ivosidenib 500mg once daily, the CR + PR rate was 39% (95% CI: 17.3-64.3) (all CR responses), with a median duration of response not estimable (range 1.9, 80.8+ months). Of the 9 patients that were TD at baseline, 6 achieved TI (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 pruritis.