How might olutasidenib long-term data impact treatment strategies in patients with IDH1-mutated R/R AML?

During the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting, the AML Hub was pleased to speak with Jorge Cortes, Augusta University, Augusta, US. We asked, How might long-term follow-up data regarding olutasidenib impact treatment strategies in patients with IDH1-mutated relapsed/refractory (R/R) acute myeloid leukemia (AML)? 

In December 2022, olutasidenib was granted  U.S. Food and Drug Administration (FDA) approval for the treatment of patients with IDH1-mutated R/R AML. Here, Cortes discusses the final 5-year efficacy and safety findings from the pivotal cohort of the phase II trial (NCT02719574) of olutasidenib in patients with IDH1-mutated R/R AML. Cortes highlights the high complete remission (CR)/CR with partial hematological recovery rate (35%), overall response rate (48%) and durable remission rates, as well as the prolonged survival and minimal toxicity of olutasidenib in patients with IDH1-mutated R/R AML including in patients who are R/R to prior venetoclax therapy. He mentions that olutasidenib is a promising treatment option for patients with R/R AML with IDH1 mutations; however, its comparison with other treatments, such as ivosidenib—which is also highly effective and commercially available—remains uncertain without a randomized trial.