Ivosidenib in relapsed or refractory acute myeloid leukemia: a profile of its use in the USA

Abstract

Ivosidenib (Tibsovo®), a first-in-class, small molecule inhibitor of mutant isocitrate dehydrogenase 1 (IDH1), is an important new therapeutic option in the USA for treating adults with relapsed or refractory acute myeloid leukemia who have a susceptible IDH1 gene mutation. In such patients, oral ivosidenib is effective and generally well tolerated, producing encouraging rates and durations of complete remission (CR), CR or CR with partial hematologic recovery (CR/CRh), overall response, and overall survival. CR/CRh rates are consistent across most baseline characteristics. Ivosidenib also provides additional clinical benefits, including transfusion independence, improvements in hematologic variables, and deep molecular responses as measured by IDH1 mutation clearance in patients who achieve CR/CRh.