August 1, 2017 — The U.S. Food and Drug Administration (FDA) has approved Enasidenib for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who carry a specific genetic mutation. The drug is approved for use in conjunction with the *RealTime IDH2* diagnostic test, a companion diagnostic kit designed to detect specific mutations in the *IDH2* gene of AML patients.
Richard Pazdur, M.D., Director of the FDA’s Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research, stated: "Enasidenib is a targeted therapy that addresses an unmet medical need for patients with relapsed or refractory AML who have an *IDH2* mutation. The use of Enasidenib was associated with complete remission in some patients and reduced the need for red blood cell and platelet transfusions."
Acute myeloid leukemia (AML) is a rapidly progressing cancer that forms in the bone marrow and results in an increased number of abnormal white blood cells in the blood and bone marrow. The National Cancer Institute, part of the National Institutes of Health, estimates that approximately 21,380 people will be diagnosed with AML this year; in 2017, approximately 10,590 patients with AML are expected to die from the disease.
Enasidenib is an isocitrate dehydrogenase-2 (*IDH2*) inhibitor that works by blocking several enzymes that promote cell growth. If an *IDH2* mutation is detected in blood or bone marrow samples using the *RealTime IDH2* test, the patient may be eligible for treatment with Enasidenib.
Common side effects of Enasidenib include nausea, vomiting, diarrhea, elevated bilirubin levels (a substance found in bile), and decreased appetite. Pregnant or breastfeeding women should not take Enasidenib, as it may cause harm to the developing fetus or newborn.
The prescribing information for Enasidenib includes a boxed warning noting that an adverse reaction known as differentiation syndrome may occur, which can be fatal if left untreated.
