All-oral Regimen Approved for Elderly Adults With Acute Myeloid Leukemia
The FDA approved oral decitabine and cedazuridine with venetoclax for newly diagnosed acute myeloid leukemia in older patients.
The U.S. Food and Drug Administration (FDA) approved oral tablets of decitabine and cedazuridine (Inqovi) in combination with venetoclax (Venclexta) for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults who are either 75 years or older or who cannot tolerate intensive induction chemotherapy due to comorbidities.
Decitabine is incorporated into DNA and induces molecular changes that can stop cell proliferation or promote cell death. Cedazuridine prevents the cell from degrading decitabine too quickly. Venetoclax is a targeted therapy that kills cancer cells by inhibiting BCL-2, a protein that blocks the processes that lead to cell death.
Older patients do not tolerate chemotherapy as well as younger patients, and for patients with AML whose health precludes treatment with intensive chemotherapy, decitabine and venetoclax is the standard of care. Previously, such treatment required intravenous administration, a route that entails travel to a health care provider. This approval marks the first all-oral version of the decitabine, cedazuridine, and venetoclax regimen for this patient population.
The approval was based on results from Study ASTX727-07, a single-arm, open-label, phase I/II clinical trial that enrolled 101 patients who had been diagnosed with AML and were either at least 75 years old or ineligible for intensive chemotherapy due to comorbidities. All patients received decitabine and cedazuridine tablets with venetoclax.
After a median of 11.2 months of follow-up, 42 patients out of 101 experienced complete remission (41.6%). The median time to complete remission was two months, and more than half of the complete remissions were ongoing at the time of follow-up.
The recommended dose of this treatment regimen is a daily oral dose of decitabine (35 mg) and cedazuridine (100 mg) on the first five days of a 28-day cycle. Oral venetoclax tablets should be given in a ramp-up phase for the first three days—100 mg on day one, 200 mg on day two, and 400 mg on day three—and continued daily at the 400 mg dose thereafter. The entire treatment should be continued until disease progression or unacceptable toxicity.
AML is a cancer of the blood and bone marrow that forms when myeloid stem cells become abnormal, immature white blood cells called myeloblasts, which crowd out healthy cells. It is the most common type of acute leukemia in adults, with most cases occurring in older adults. According to federal statistics, it was estimated that 22,720 individuals would be diagnosed with acute myeloid leukemia and 11,500 patients would die of the disease in the United States in 2026.
The FDA rendered its decision on May 13, 2026. Check this resource for updated information on all therapeutics regulated by the FDA.