FDA approves Agios’ Tibsovo for acute myeloid leukemia treatment
Tibsovo (ivosidenib) developed by Agios Pharmaceuticals has been approved by the US Food and Drug Administration (FDA) for acute myeloid leukemia treatment in patients with a specific genetic mutation and whose condition relapsed or is refractory.
The Agios AML drug has been indicated to be used with an FDA-approved companion diagnostic that will detect specific mutations in the IDH1 gene in AML patients.
According to the FDA, AML is a rapidly progressing cancer that develops in the bone marrow. AML leads to a large number of abnormal white blood cells in the bloodstream and bone marrow.
An isocitrate dehydrogenase-1 inhibitor, Tibsovo works in acute myeloid leukemia treatment by reducing abnormal creation of the oncometabolite 2-hydroxyglutarate (2-HG), thereby leading to separation of malignant cells.
The FDA said that if the IDH1 mutation is detected in blood or bone marrow samples via an FDA-approved test, then the patient could be eligible for acute myeloid leukemia treatment with Tibsovo. The regulator has also approved the RealTime IDH1 Assay, a companion diagnostic that can detect the IDH1 mutation.
Commenting on Tibsovo or Ivosidenib FDA approval for acute myeloid leukemia treatment, Richard Pazdur – director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said: “Tibsovo is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH1 mutation.
“The use of Tibsovo is associated with a complete remission in some patients and a reduction in the need for both red cell and platelet transfusions.”
Tibsovo FDA approval was based on the results of a single-arm trial of 174 adult patients having relapsed or refractory AML with an IDH1 mutation.
The trial evaluated gauged the percentage of patients with no evidence of disease and full recovery of blood counts after acute myeloid leukemia treatment, defined as complete remission or CR. It also measured percentage of patients with no evidence of disease and partial recovery of blood counts following Tibsovo treatment, defined as complete remission with partial hematologic recovery or CRh.
With a median follow-up lasting 8.3 months, 32.8% of patients on acute myeloid leukemia treatment with Tibsovo registered a CR orCRh that lasted a median 8.2 months. Out of the 110 patients who needed transfusions of blood or platelets owing to AML at the launch of the trial, 37% went at least 56 days without needing a transfusion after Tibsovo therapy.