FDA approves targeted treatment for patients with relapsed or refractory AML who have a certain genetic mutation

FDA approved on Friday ivosidenib (Tibsovo—Agios Pharmaceuticals) tablets for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) who have a particular genetic mutation. The drug is approved for use with an FDA companion diagnostic used to identify specific mutations in the IDH1 gene in patients who have AML.

FDA approved on Friday ivosidenib (Tibsovo—Agios Pharmaceuticals) tablets for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) who have a particular genetic mutation. The drug is approved for use with an FDA companion diagnostic used to identify specific mutations in the IDH1 gene in patients who have AML. FDA also approved the diagnostic, the RealTime IDH1 Assay (Abbott Laboratories), on Friday. "Tibsovo is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH1 mutation," said Richard Pazdur, MD, director of FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research. "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." Common adverse effect associated with ivosidenib include fatigue increase in white blood cells, joint pain, diarrhea, shortness of breath, swelling in the arms or legs, nausea, pain or sores in the mouth or throat, QT prolongation, rash, fever, cough, and constipation. The drug must be dispensed with a patient Medication Guide detailing key information about the product's uses and risks. The prescribing information also includes a boxed warning about an adverse reaction known as differentiation syndrome that can occur and be fatal if not treated.