FDA Approves Celgene’s AML Treatment Paired with Abbott’s Companion Diagnostic

Patients with acute myeloid leukemia (AML), an aggressive form of blood cancer, could now have a new treatment option thanks to the US Food and Drug Administration’s (FDA) approval of Celgene’s Idhifa (enasidenib). The RealTime IDH2 Assay, a companion diagnostic developed by Abbott Laboratories, is used to identify cancer patients with an IDH2 mutation who may benefit from therapy with Idhifa.

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

Patients with AML accumulate abnormal white blood cells in their bloodstream and bone marrow. These abnormal leucocytes rapidly outnumber normal cells, compromising a patient’s ability to fight infection.

Over 21,000 new patients will be diagnosed with AML in 2017, according to the National Cancer Institute at the National Institutes of Health. More than 10,000 patients with AML will die of the cancer this year alone.

Treatment regimens for AML still rely on two chemotherapeutic agents that are highly toxic in adults and aren’t effective for all patients. Less than 20 percent of patients aged 60 years and older survive for five years or more after receiving their initial AML diagnosis.

Approximately 12 percent of patients diagnosed with AML carry a mutation in the IDH2 gene. Patients with relapsed or refractory AML that are found to carry this mutation, based on the RealTime IDH2 Assay companion diagnostic, could be eligible for treatment with Idhifa.

“AML patients desperately need new and better options for treatment,” said Louis J. DeGennaro, President and CEO of The Leukemia & Lymphoma Society. “For too long, we’ve treated AML as a one-size-fits-all disease and we are changing that.”