FDA Approves New Targeted Therapies for Acute Myeloid Leukemia


The U.S. Food and Drug Administration (FDA) has signed off on a new targeted treatment for some patients with acute myeloid leukemia (AML), an often aggressive disease and the most common adult acute leukemia.

Leukemia cells.

The FDA approved the drug Idhifa (enasidenib) specifically for the treatment of adult patients with relapsed or refractory AML who have IDH2 genetic mutations. IDH2 mutations are found in about 12 percent of AML patients. The approval follows FDA’s approval earlier this year of midostaurin (Rydapt®) for newly diagnosed AML patients with a mutation to the gene FLT3.

Richard Stone, oncologist, leukemia

Richard Stone, MD

Idhifa is a big step toward addressing the unique needs of these particular AML patients and achieving better outcomes, according to Richard Stone, MD, program director of the adult leukemia program at Dana-Farber. It can also help improve quality of life, as the new treatment may mean many of these patients no longer need regular red cell and platelet transfusions. Stone and his colleagues at Dana-Farber/Harvard Cancer Center enrolled a large number of patients in the clinical trial which led to the approval of this agent.

Idhifa was studied in a trial of 199 patients with relapsed or refractory AML and an IDH2 mutation. After a minimum of six months of treatment, 19 percent of patients experienced complete remission for a median of 8.2 months, and four percent of patients experienced complete remission with partial hematologic recovery for a median of 9.6 months. Thirty-four percent of the 157 patients who needed blood or platelet transfusions because of their AML no longer needed those transfusions after their treatment with Idhifa.

Midostaurin was approved as a combination treatment with chemotherapy for adult patients newly diagnosed with AML that carries a mutation in the gene FLT3. Stone led clinical testing for the drug, which became the first new treatment for newly diagnosed acute myeloid leukemia (AML) in more than 25 years.

Learn more about acute myeloid leukemia (AML) from the adult leukemia program at Dana-Farber.

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