FDA approves targeted acute myeloid leukemia treatment

SILVER SPRING, Md. — The Food and Drug Administration has approved a new, targeted treatment for patients with relapsed/refractory acute myeloid leukemia. Idhifa (enasidenib), from Celgene, was approved for use with the RealTime IDH2 Assay, used to detect specific mutations in the IDH2 gene in patients with acute myeloid leukemia.

“Idhifa is a targeted therapy that fills an unmet 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. “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.”

AML is a form of cancer that progresses rapidly, forming in bone marrow and resulting 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 roughly 21,380 people will be diagnosed with the disease this year, and estimates that it will kill 10,590 patients in 2017.

We appreciate the FDA’s efforts to expedite the availability of IDHIFA for patients with this devastating disease weeks ahead of the PDUFA date,” said Mark Alles, Chief Executive Officer of Celgene. “This milestone further illustrates the value of Celgene’s unique distributed research model. Our partnership with Agios is an exceptional example of how Celgene and its collaborators can positively impact the lives of patients with high unmet needs.”