The Food and Drug Administration (FDA) has expanded the indication for Tibsovo (ivosidenib; Agios) to include treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adult patients who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy. Previously, the treatment had been approved only for relapsed or refractory AML with a susceptible IDH1 mutation.
The approval was based on data from an open-label, single-arm study that included 28 adult patients with newly diagnosed AML with an IDH1 mutation. The primary endpoint of the study was the combined complete remission (CR) and complete remission with partial hematologic improvement (CRh) rate.
Treatment with Tibsovo resulted in a CR rate of 28.6%, a CRh rate of 14.3%, and a CR+CRh rate of 42.9%; 58.3% of patients who achieved CR or CRh were in remission at 1 year after receiving treatment. For patients who achieved a CR or CRh, the median time to CR or CRh was 2.8 months (range: 1.9 to 12.9 months).
Among patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline (N=17), 41.2% became independent of RBC and platelet transfusions during any 56-day post-baseline period. Of the patients who were independent of both RBC and platelet transfusions at baseline (N=11), 54.5% remained transfusion independent during any 56-day post-baseline period.
“The phase 1 results for Tibsovo demonstrated that this oral, single agent therapy can induce durable responses in newly diagnosed AML patients with an IDH1 mutation,” said Gail J. Roboz, MD, Professor of Medicine, Director of the Leukemia Program and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine and NewYork-Presbyterian/Weill Cornell Medical Center. “Many patients included in the study had features associated with particularly aggressive and challenging forms of AML, including secondary disease, adverse risk genetics and prior treatment with hypomethylating agents.”
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