Olutasidenib granted FDA approval for patients with IDH1-mutated R/R AML

On December 1, 2022, olutasidenib, an oral, small molecule, IDH1 inhibitor was approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with IDH1-mutated relapsed or refractory (R/R) acute myeloid leukemia (AML).¹

This approval is based on results from a phase II study (NCT02719574) that has previously been reported on by the AML Hub. Briefly, the study included 147 adult patients with R/R AML who were treated with oral olutasidenib at a dose of 150 mg, twice daily.¹

• A total of 35% of patients achieved either complete remission (CR) or complete remission with partial hematological recovery (CRh).¹
• The median time to CR+CRh was 1.9 months and the median duration of CR+CRh was 25.9 months.¹
• In 86 patients who were red blood cell and/or platelet dependent at baseline, 34% became transfusion independent.¹
• In 61 patients who were transfusion independent at baseline, 64% remained transfusion independent during any 56-day post-baseline period.¹

Olutasidenib was well tolerated with an acceptable safety profile. The most common adverse reactions included nausea, fatigue/malaise, arthralgia, constipation, leukocytosis, dyspnea, fever, rash, mucositis, diarrhea, and transaminitis. Olutasidenib has a boxed warning regarding the risk of differentiation syndrome.¹