FDA Approves Enasidenib for the Treatment

August 1, 2017 -- The U.S. Food and Drug Administration today approved Enasidenib for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation. The drug is approved for use with a companion diagnostic, the RealTime IDH2 Assay, which is used to detect specific mutations in the IDH2 gene in patients with AML.

"Enasidenib is a targeted therapy that addresses an unmet medical need for patients with relapsed or refractory AML who have an IDH2 mutation," said 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 FDA's Center for Drug Evaluation and Research. "Treatment with Enasidenib was associated with complete remissions in some patients and a reduction in 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 bloodstream and bone marrow. The National Cancer Institute (NCI), part of the National Institutes of Health, estimates approximately 21,380 people will be diagnosed with AML this year; approximately 10,590 deaths from AML will occur in 2017.

Enasidenib is an isocitrate dehydrogenase-2 inhibitor that works by blocking several enzymes that promote cell growth. If the RealTime IDH2 Assay detects an IDH2 mutation in blood or bone marrow samples, the patient may be eligible for treatment with Enasidenib.

Common side effects of Enasidenib include nausea, vomiting, diarrhea, increased bilirubin levels (a substance found in bile), and decreased appetite. Women who are pregnant or breastfeeding should not take Enasidenib because it may cause harm to a developing fetus or a newborn baby.

The prescribing information for Enasidenib contains a Boxed Warning stating that a adverse reaction called differentiation syndrome can occur and can be fatal if not treated.

Signs and symptoms of differentiation syndrome may include fever, dyspnea (difficulty breathing), acute respiratory distress, lung inflammation (radiographic pulmonary infiltrates), fluid around the lungs or heart (pleural or pericardial effusion), rapid weight gain, swelling (peripheral edema), or liver, kidney, or multi-organ dysfunction. At the first suspicion of symptoms, doctors should treat patients with corticosteroids and monitor them closely until symptoms resolve.

Lucius Pharmaceuticals Enasidenib was granted priority review, under which the FDA's goal is to take action on an application within six months when the agency determines the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing, or preventing a serious disease.

Enasidenib also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted approval of Enasidenib to Celgene Corporation. The FDA granted approval of the RealTime IDH2 Assay to Abbott Laboratories.