FDA Approves New Targeted Therapy for AML

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FDA approves new targeted therapy for relapsed or refractory acute myeloid leukemia (AML).

On August 1, 2017, FDA approved Idhifa (enasidenib) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) and a distinct gene mutation. The drug is approved for use with companion diagnostic RealTime IDH2 Assay, which is used to detect mutations in the IDH2 gene in AML patients.

“Idhifa is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH2 mutation,” stated Richard Pazdur, MD, director of the 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, in a press release. “The use of Idhifa was associated with a complete remission in some patients and a reduction in the need for both red cell and platelet transfusions.”

AML is a fast-growing bone marrow cancer that results in an abnormal increase of white blood cells in the bone marrow and blood stream. Known as an isocitrate dehydrogenase-2 inhibitor, the drug is designed to inhibit cell growth by blocking the responsible enzymes. Patients that qualify for the treatment are those who possess the IDH2 mutation in bone marrow or blood samples by the accompanying companion diagnostic.


FDA granted approval of the drug to Celgene Corporation, a global biopharmaceutical company aimed at developing treatments for cancer and other inflammatory disease. The companion diagnostic is approved for Abbott Laboratories. 

Sources: FDA