FDA Expands Approval of Ivosidenib and Azacitidine for IDH1-Mutant Newly Diagnosed AML

Patients with acute myeloid leukemia who are 75 years or older or ineligible for induction chemotherapy due to comorbidities can now receive treatment with ivosidenib and azacitidine.

A combination of ivosidenib (Tibsovo) and azacitidine (Onureg) have received approval by the FDA for the treatment of patients with newly diagnosed IDH-1 mutant acute myeloid leukemia (AML) for those who are 75 years or older or with comorbidities that prevent the use of induction chemotherapy.

The expansion was based on findings from the phase 3 AGILE trial (NCT03173248), assessing the combination vs placebo in IDH1-mutant AML, which highlighted a statistically significant improvement in event-free survival (HR, 0.35; 95% CI, 0.17-0.72; 2-sided P = .0038) and overall survival (OS; HR, 0.44; 95% CI, 0.27-0.73; 2-sided P = .0010) in the experimental arm. Additionally, the median OS was 24 months compared with the placebo arm at 7.9 months.

“Today’s approval builds on the established body of evidence for [ivosidenib], which is now approved across multiple IDH1-mutated cancer types,” David K. Lee, chief executive officer at Servier Pharmaceuticals, said in a press release. “As a leader in oncology pioneering the science behind targeted IDH inhibition, we are proud to bring a new therapeutic option to the acute myeloid leukemia community and remain committed to pushing the boundaries of healthcare innovation in oncology and beyond.”

Ivosidenib received priority review by the FDA in March 2022 for IDH1-positive AML.


Servier Announces FDA Approval of TIBSOVO® (ivosidenib tablets) in combination with azacitidine for patients with newly diagnosed IDH1-mutated acute myeloid leukemia. News release. Servier. May 25, 2022. Accessed May 25, 2022. https://prn.to/3wMibQh