Recently, enasidenib and ivosidenib, oral inhibitors of mutated IDH2 and IDH1 genes, respectively, were approved for use in relapsed or refractory acute myeloid leukemia.
The FDA approved ivosidenib (Tibsovo; Agios), a small-molecule inhibitor of isocitrate dehydrogenase (IDH)1 on July 20, 2018, for treatment of adults with relapsed or refractory acute myeloid leukemia (R/R AML) with susceptible IDH1 mutation as detected by an FDA-approved test.
This article summarizes the milestones in the development of ivosidenib leading to this first approval in the USA for the treatment of patients with relapsed or refractory AML with a susceptible IDH1 mutation.
In patients with advanced IDH1-mutated relapsed or refractory AML, ivosidenib at a dose of 500 mg daily was associated with a low frequency of grade 3 or higher treatment-related adverse events and with transfusion independence, durable remissions, and molecular remissions in some patients with complete remission.
The experimental drug AG-120, which inhibits mutant IDH1, showed promising results in a phase I trial in patients with relapsed or refractory acute myeloid leukemia.