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First Targeted Treatment Approved for Relapsed and Refractory Acute Myeloid Leukemia
By Krista Rossi

The US Food and Drug Administration (FDA) has approved the first drug in its class (IDH1 inhibitors), ivosidenib (Tibsovo), for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation. The drug us also approved for use with an FDA-approved companion diagnostic to detect specific mutations in the IDH1 gene in patients with AML.

“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 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, in a recent statement. “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.”

Ivosidenib is an isocitrate dehydrogenase-1 inhibitor that works by decreasing abnormal production of the oncometabolite 2-hydroxyglutarate (2-HG), leading to differentiation of malignant cells. A patient may be eligible for ivosidenib treatment if, by using an FDA-approved test, the IDH1 mutation is detected in blood or bone marrow samples. The mutation can be detected with the RealTime IDH1 Assay, a companion diagnostic also approved today.


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