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How does Idhifa work in acute myeloid leukemia (AML)?

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Oct 4, 2020.

Official Answer

by Drugs.com

Idhifa (enasidenib) is an oral targeted treatment for acute myeloid leukemia (AML) that works by blocking the enzyme isocitrate dehydrogenase-2 (IDH2). This helps young red blood cells (blasts) in your bone marrow to mature into healthy and functioning red and white blood cells and platelets. It also helps to relieve overcrowding of immature blood cells in the marrow.

These cells are needed in our bodies for oxygen delivery to tissues (red blood cells), to help fight infection (white blood cells), and help prevent bleeding (platelets).

In August 2017, the FDA approved Celgene's Idhifa for the treatment of adult patients with acute myeloid leukemia (AML) with an IDH2 mutation whose disease has come back or has not improved after previous treatments. About 8% to 19% of people with AML have the IDH2 mutation.

Idhifa was the first medication in a medication class called IDH2 inhibitors. It targets the mutant IDH2 variants R140Q, R172S, and R172K.

How well does Idhifa work in AML?

The effectiveness of Idhifa was demonstrated in a single arm trial of 199 patients with relapsed or refractory AML who had IDH2 mutations as detected by the RealTime IDH2 Assay.

  • After at least 6 months of treatment, 19% of patients experienced complete remission (no evidence of disease and full recovery of blood counts after treatment) for a median time of 8.2 months. In addition, 4% of patients experienced complete remission with partial recovery of blood count for a median time of 9.6 months.
  • 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.
  • Common side effects with Idhifa include nausea, vomiting, diarrhea, increased levels of bilirubin (substance found in bile) and decreased appetite.

Bottom Line

  • Idhifa (enasidenib) is an oral targeted treatment for acute myeloid leukemia (AML) that works by blocking the enzyme isocitrate dehydrogenase-2 (IDH2).
  • Blocking IDH2 helps young red blood cells (blasts) in your bone marrow to mature into healthy and functioning red and white blood cells and platelets.
  • These cells are needed in our bodies for oxygen delivery to tissues, to help fight infection, and help prevent bleeding.

This is not all the information you need to know about Idhifa for safe and effective use. Review the full Idhifa (enasidenib) information here, and discuss this information with your doctor or other health care provider.

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