FDA Approves Oral Decitabine/Cedazuridine and Venetoclax for AML

The treatment landscape for older or medically frail patients with acute myeloid leukemia (AML) is shifting toward oral regimens, offering oncology nurses new opportunities for outpatient management and patient education.

On May 13, the Food and Drug Administration (FDA) approved decitabine and cedazuridine (Inqovi) tablets in combination with venetoclax (Venclexta) for adults newly diagnosed with AML who are aged 75 years or older or have comorbidities that preclude the use of standard intensive induction chemotherapy.

Clinical trial outcomes

This approval was supported by results from Study ASTX727-07, a single-arm, open-label clinical trial involving 101 adult patients with newly diagnosed AML who were ineligible for intensive chemotherapy. Data from the trial indicated that 41.6% of participants achieved complete remission (CR).

The median time to reach CR was two months (range, 0.4–15.3 months). While the median duration of complete remission (DoCR) was not reached during the study, the observed durations ranged from 0.5 to 16.3 months.

This oral pathway represents a significant shift from standard treatment, which often requires lengthy hospital stays and carries high risks of severe side effects for older or frail populations.

Administration and dosing

The newly approved regimen utilizes a tablet form of decitabine and cedazuridine to simplify administration. The recommended dosage for Inqovi, when used in conjunction with venetoclax, is one tablet (35 mg decitabine/100 mg cedazuridine) taken daily on Days 1 through 5 of each 28-day cycle. Patients should continue this schedule until disease progression or the occurrence of unacceptable toxicity.

Safety and nursing considerations

Oncology nurses should be vigilant in monitoring for myelosuppression, a primary safety concern noted in the prescribing information. Myelosuppression can lead to decreased bone marrow activity, resulting in reduced levels of red blood cells, white blood cells, and platelets. Furthermore, the treatment carries a warning for embryo-fetal toxicity, and healthcare professionals are encouraged to report any serious adverse events to the FDA’s MedWatch Reporting System.

Global collaboration

The review was conducted under Project Orbis, an initiative by the FDA Oncology Center of Excellence that facilitates concurrent submission and review of oncology products with international partners. For this approval, the FDA collaborated with Health Canada. Full prescribing information is available through the Drugs@FDA database.

Reference

1. FDA approves oral combination of decitabine and cedazuridine tablets with venetoclax for newly diagnosed acute myeloid leukemia. News release. FDA. May 13, 2026.