The U.S. Food and Drug Administration has approved Idhifa (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 patients with relapsed or refractory AML who have specific mutations in the IDH2 gene.
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 at the National Institutes of Health estimates that approximately 21,380 people will be diagnosed with AML this year; approximately 10,590 patients with AML will die of the disease in 2017.
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 a companion diagnostic, the RealTime IDH2 Assay, the patient may be eligible for treatment with Idhifa.
The efficacy of Idhifa was studied in a single-arm trial of 199 patients with relapsed or refractory AML who had IDH2 mutations. Of the 157 patients who required transfusions of blood or platelets due to AML at the start of the study, 34 % no longer required transfusions after 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.
https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm569421.htm
