New targeted treatment for relapsed or refractory acute myeloid leukemia approved by FDA

Idhifa (enasidenib) which is used for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) has been approved by the U.S. food and Drug Administration.

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 said, "Idhifa is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH2 mutation. 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."

Idhifa 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 Idhifa.

Idhifa was granted Priority Review designation, under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition. Idhifa also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

FDA granted the approval of Idhifa was granted to Celgene Corporation and RealTime IDH2 Assay to Abbott Laboratories.