FDA Approves Tibsovo Combination for Older Patients with Leukemia

Tibsovo is the first therapy targeting cancer metabolism for patients with newly diagnosed IDH1-mutated acute myeloid leukemia.

The FDA has approved Servier Pharmaceuticals’ Tibsovo (ivosidenib) in combination with azacitidine for the treatment of patients with newly diagnosed IDH1-mutated acute myeloid leukemia (AML). It is indicated for adults 75 years or older or those who are ineligible for intensive induction chemotherapy.

Tibsovo is the first therapy targeting cancer metabolism approved in combination with azacitidine for patients with newly diagnosed IDH1-mutated AML.

AML is a difficult-to-treat cancer of the blood and bone marrow and is one of the most common types of leukemia in adults with about 20,000 new cases estimated in the United States each year. The IDH1 gene provides instructions for making the enzyme isocitrate dehydrogenase 1, which produces a molecule necessary for cellular processes. IDH1 mutations are present in about 6% to 10% of AML cases.

“People living with acute myeloid leukemia, especially those who are newly diagnosed and are not eligible for intensive chemotherapy, have had few treatment options,” Susan Pandya, M.D., vice president clinical development and head of cancer metabolism global development oncology and immuno-oncology at Servier.

The approval was based on the phase 3 AGILE trial, which demonstrated improvement in event-free survival and overall survival. Tibsovo plus azacitidine resulted in a threefold improvement in median overall survival (24 months) compared with placebo plus azacitidine (7.9 months) as a first-line treatment for IDH1-mutated AML. Results from the AGILE study were presented at the 2021 American Society of Hematology (ASH) Annual Meeting and Exposition, and recently published in the New England Journal of Medicine.

Tibsovo is also approved as monotherapy for the treatment of adults with IDH1-mutant relapsed or refractory AML, and for adults with newly diagnosed IDH1-mutated AML who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy. Last year, the FDA approved Tibsovo for its first approval in a non-hematologic malignancy for patients with previously treated IDH1-mutated cholangiocarcinoma