FDA approves the frontline use of ivosidenib in AML patients with IDH1 mutation

June 2022 Pharma News Nalinee Pathak

The Food and Drug Administration (FDA) has approved the use of ivosidenib (Tibsovo) plus azacitidine for acute myeloid leukaemia (AML) with a susceptible IDH1 mutation. The approval for this drug has been granted based on clinical study-AG120-C-009, NCT03173248.

Study Details

The double-blind, multicentre, placebo-controlled study enrolled 146 newly diagnosed AML patients with a mutation in the IDH1 gene who were randomised (1:1) to receive either ivosidenib (500mg, daily, n=74) or matched placebo (n=74, once daily), on Days 1-28 in combination with azacitidine 75 mg/m2/day on Days 1-7 or Days 1-5 and 8-9 of each 28-day cycle until disease progression. Only those participants were included who met at least one of the following criteria-age 75 years or older, baseline Eastern Cooperative Oncology Group performance status of 2, severe cardiac or pulmonary disease, hepatic impairment with bilirubin > 1.5 times the upper limit of normal, creatinine clearance < 45 mL/min, or another comorbidity.


The median overall survival was prolonged in the ivosidenib plus azacitidine arm to 24.0 months (95% CI: 11.3, 34.1) than as compared to the 7.9 months (95% CI: 4.1, 11.3) in the placebo + azacitidine control arm (HR 0.44; 95% CI: 0.27, 0.73; p=0.0010). A higher complete remission was observed in the ivosidenib arm versus the control arm (47% vs 15%). Finally, event-free survival events occurred in 65% of the combination group and 84% in the control group. The most common adverse events included diarrhoea, fatigue, edema, nausea and vomiting.


Based on the efficacy and safety profile of ivosidenib (Tibsovo), it has received the FDA nod for usage in combination with azacitidine in AML patients with IDH1 mutation.


Press release by the FDA