The U.S. Food and Drug Administration (FDA) has approved ivosidenib as the first IDH1 inhibitor for patients with relapsed or refractory acute myeloid leukemia (AML) and an IDH1 mutation. Ivosidenib was approved simultaneously with the RealTime IDH1 Assay, a companion diagnostic that is used to detect the IDH1 mutation.
The FDA's approval of ivosidenib was based on results from a single-arm trial of 174 adults with relapsed or refractory AML with an IDH1 mutation. After a median follow-up of 8.3 months, 32.8 percent of patients experienced a complete remission (CR) or a CR with partial hematologic recovery (CRh). The median duration of CR or CRh was 8.2 months. Of the 110 patients who were transfusion-dependent at baseline, 37 percent went at least 56 days without requiring a transfusion after treatment with ivosidenib.
Common adverse events associated with ivosidenib included fatigue, leukocytosis, arthralgia, diarrhea, shortness of breath, peripheral edema, nausea, pain in the mouth or throat, irregular heartbeat, rash, fever, cough, and constipation.
Source: FDA press release, July 20, 2018.