FDA Approves Olutasidenib for Acute Myeloid Leukemia Subtype

The FDA’s approval of oral olutasidenib provides adults with relapsed or refractory acute myeloid leukemia harboring a susceptible IDH1 mutation a new treatment option.

The FDA approved olutasidenib for treating patients with relapsed or refractory acute myeloid leukemia susceptible to harboring an IDH1 mutation, according to a press release from the FDA.

The FDA has also approved the Abbott RealTime IDH1 Assay, which is used to select patients suitable for receiving olutasidenib.

The FDA based its approval on findings from the single-arm Study 2102-HEM-101 (NCT02719574) clinical trial. Investigators of the trial reported that the rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 35% (95% CI, 27%-43%) among patients receiving olutasidenib, which included a 32% CR rate and 2.7% CRh.

The median time to CR plus CRh was 1.9 months (range, 0.9-5.6), and the median duration of CR+CRh among patients was 25.9 months (95% CI, 13.5-not reached [NR]).

Among 86 patients who received red blood cell or platelet transfusions at baseline, 34% (n = 29) became independent of these transfusions 56 days after baseline. Of 61 patients who were independent of both red blood cell and platelet transfusions at baseline, 64% (39) remained transfusion independent after 56 days following baseline.

Patients in the study received 150 mg of oral olutasidenib twice a day and continued treatment until disease progression, unacceptable toxicity, or hematopoietic stem cell transplantation. The median treatment duration was 4.7 months (range, 0.1-26), and 11% of patients underwent hematopoietic stem cell transplantation following receipt of study drug.

The most common adverse effects observed in at least 20% of patients during the study included nausea, fatigue or malaise, arthralgia, constipation, leukocytosis, dyspnea, fever, rash, mucositis, diarrhea, and transaminitis.


FDA approves olutasidenib for relapsed or refractory acute myeloid leukemia with a susceptible IDH1 mutation. News release. FDA. December 1, 2022. Accessed December 1, 2022. bit.ly/3XRAW06