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FDA Expands Approval of Tibsovo for Treatment of AML with IDH1 Mutation

The U.S. Food and Drug Administration (FDA) has expanded the approval of Tibsovo® (ivosidenib), a targeted therapy, for newly diagnosed acute myeloid leukemia (AML) with an IDH1 mutation in people who are at least 75 years old or with comorbidities that prevent the use of intensive chemotherapy. The IDH1 mutation is detected through an FDA-approved test.1

Tibsovo was approved in July 2018 as the first targeted treatment for people with AML that has relapsed or is refractory (meaning that the cancer hasn’t responded to previous treatment or has stopped responding).

Approval based on research

The expansion of Tibsovo was based on results from a Phase I open-label, single-arm, multi-center clinical trial of 28 patients either aged 75 or older or with comorbidities that prohibit them from receiving induction chemotherapy, which is an intensive first phase of chemotherapy. 42.9% of patients in the study achieved either complete remission or complete remission with partial hematologic improvement, which was defined as “defined as less than 5% of blasts in bone marrow, no evidence of disease and partial recovery of peripheral blood counts.”2

AML with an IDH1 mutation

AML develops due to mutations in the myeloid cells, which are the cells that normally develop into red blood cells, platelets, and white blood cells. Roughly 6% to 10% of people with AML have a mutation on the isocitrate dehydrogenase-1 (IDH1) gene.3 Tibsovo is a targeted therapy that specifically works on AML that has IDH1 mutation, which is detected through an FDA-approved genetic test.

  1. FDA approves ivosidenib as first-line treatment for AML with IDH1 mutation. U.S. Food & Drug Administration. Available at Accessed May 3, 2019.
  2. FDA expands approval of Tibsovo for IDH1-mutant acute myeloid leukemia. Healio. Available at Accessed May 3, 2019.
  3. Sagonowsky E. Agios wins FDA nod for targeted AML drug Tibsovo. Fierce Pharma. Available at Accessed May 7, 2019