Ivosidenib granted approval by the European Commission for the treatment of adult patients with IDH1-mutated AML

On May 10, 2023, oral ivosidenib, a small molecular isocitrate dehydrogenase-1 (IDH1) inhibitor, was approved by the European commission in combination with azacitidine for the treatment of adult patients with newly diagnosed (ND) acute myeloid leukemia (AML) with an IDH1^R132 mutation who are ineligible for standard induction chemotherapy.¹

Following key data from the phase III AGILE trial (NCT03173248), ivosidenib in combination with azacitidine is already approved by the U.S Food and Drug Administration (FDA) for the treatment of IDH1-mutated AML in adult patients ≥75 years old and for those with comorbidities precluding the use of standard intensive induction chemotherapy.² Results demonstrated a favorable safety profile,  improved event-free survival (hazard ratio [HR], 0.33; 95% confidence interval [CI], 0.16, 0.69), and improved overall survival (HR, 0.44; 95% CI, 0.27, 0.73) in patients with IDH1-mutated ND AML who received ivosidenib plus azacitidine, compared with those who received azacitidine plus placebo.¹

Ivosidenib is also FDA approved as a monotherapy in patients with relapsed/refractory or ND IDH1-mutated AML³, and in patients with locally advanced or metastatic IDH1-mutated cholangiocarcinoma who have been previous treated.^1,3