New Treatment for Acute Myeloid Leukemia Approved by FDA

The medication is to be taken orally once daily, and works by disrupting a cellular protein that drives leukemia growth.
Nov. 25, 2025

A new treatment for acute myeloid leukemia, the deadliest blood cancer, has been approved by the FDA.

The drug ziftomenib has specifically been approved for “patients with recurring or treatment-resistant acute myeloid leukemia who have a mutation in the NPM1 gene. The new medication, taken by mouth once daily, offers a potential treatment for patients who otherwise have no good options.”

Acute myeloid leukemia is primarily seen in people over age 68. Over 22,000 Americans develop the condition each year, representing about 1 in 3 cases of blood cancer, and “more than 11,000 die.” The drug works by “disrupting interactions of a cellular protein, menin, that drive[s] leukemia cell growth and survival. It allows cells to mature into white blood cells instead of becoming cancerous.”

Trials are ongoing to determine the drug’s potential to “work in combination with other treatments to target both leukemia and solid tumors.”

About the Author

Matt MacKenzie

Associate Editor

Matt is Associate Editor for Healthcare Purchasing News.

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