Withdrawal of European Marketing Association application for ivosidenib for patients with R/R IDH1-mutated AML

On October 16, 2020, the European Marketing Association application for ivosidenib for adult patients with relapsed/refractory (R/R) IDH1-mutated acute myeloid leukemia (AML) was withdrawn. This decision was based on the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) assessment that the data from the single-arm, uncontrolled phase I study in the R/R AML setting (NCT02074839) did not sufficiently support a positive benefit–risk balance.¹

Complete remission or complete remission with partial hematologic recovery was 30.4%, with a median duration of response of 8.2 months. The overall response rate was 41.6%, with a median duration of response of 6.5 months. Treatment-related adverse events of Grade ≥ 3 included prolongation of the QT interval (7.8%), IDH differentiation syndrome (3.9%), anemia (2.2%), thrombocytopenia (3.4%), and leukocytosis (1.7%).² 

Find out more about this trial and the FDA approval here. The phase III studies of ivosidenib treatment for newly diagnosed AML, AGILE (NCT03173248) and HOVON150/AMLSG29 (NCT03839771), remain ongoing.¹