FDA approves new targeted treatment for relapsed or refractory AML – APhA submits compounding comments to FDA. – (866) 348-2889
FDA on August 1 approved enasidenib (Idhifa—Celgene) for the treatment of adults 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. The drug “is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH2 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 (enasidenib) was associated with a complete remission in some patients and a reduction in the need for both red cell and platelet transfusions.” Enasidenib is an isocitrate dehydrogenase-2 inhibitor that works by blocking several enzymes that promote cell growth. If the IDH2 mutation is detected in blood or bone marrow samples using the RealTime IDH2 Assay, the patient may be eligible for treatment with enasidenib. Common adverse events of enasidenib include nausea, vomiting, diarrhea, increased levels of bilirubin, and decreased appetite.