FDA Approves Oral Decitabine and Cedazuridine Plus Venetoclax for Newly Diagnosed Acute Myeloid Leukemia
Clinical Summary:
- FDA approved decitabine and cedazuridine plus venetoclax for adults with newly diagnosed AML who are ineligible for intensive chemotherapy, marking the first fully oral regimen for this patient population.
- Approval was based on the phase 2b ASCERTAIN-V trial, in which the regimen achieved a 46.5% complete response rate, a 63.4% CR/CRi rate, and a median overall survival of 15.5 months.
- The safety profile was consistent with known toxicities of hypomethylating agents and venetoclax, with febrile neutropenia, anemia, and neutropenia among the most common grade ≥3 adverse events; no new safety signals were identified.
On May 13, 2026, the US Food and Drug Administration (FDA) approved decitabine and cedazuridine tablets in combination with venetoclax for adults with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy.
Decitabine and cedazuridine (Inqovi; Taiho Oncology) plus venetoclax (Venclexta; AbbVie and Genentech) is the first all-oral regimen approved for this patient population, offering a potential alternative to infusion-based hypomethylating therapy for older adults and patients with significant comorbidities.
The approval was supported by findings from the phase 2b ASCERTAIN-V trial, which evaluated the regimen in 101 adults with newly diagnosed AML who were considered unsuitable for standard intensive chemotherapy. Patients received oral decitabine and cedazuridine on days 1 through 5 of each 28-day cycle together with daily oral venetoclax.
Investigators reported that the regimen produced a complete response (CR) rate of 46.5%. The combined CR and complete response with incomplete hematologic recovery (CRi) rate was 63.4%. Median overall survival reached 15.5 months, and more than 75% of patients achieving CR remained in remission at 12 months. The median time to CR was approximately 2.4 months.
Responses were observed across multiple patient subgroups, including older adults and patients with poor performance status. Researchers also reported that pharmacokinetic analyses showed no clinically meaningful drug-drug interactions between decitabine and cedazuridine and venetoclax, supporting administration in the outpatient setting.
The safety profile of the regimen was generally consistent with the known toxicities associated with hypomethylating agents and venetoclax. Grade 3 or higher adverse events were common, with febrile neutropenia, anemia, and neutropenia reported most frequently. Investigators noted that no new safety signals were identified during the study.
The approval introduces a fully oral treatment option that may help reduce the burden of frequent clinic visits and infusion appointments for patients unable to tolerate intensive chemotherapy. Clinicians say the regimen may expand access to treatment for patients who prefer outpatient care or live farther from specialized cancer centers.
Sources:
- US Food and Drug Administration. FDA approves oral combination of decitabine and cedazuridine tablets with venetoclax for newly diagnosed acute myeloid leukemia. FDA. Published February 25, 2026. Accessed May 13, 2026. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-oral-combination-decitabine-and-cedazuridine-tablets-venetoclax-newly-diagnosed-acute
- Zeidan AM, Griffiths EA, Dinardo CD, et al. An all-oral regimen of decitabine-cedazuridine plus venetoclax in patients with newly diagnosed acute myeloid leukemia ineligible for intensive induction chemotherapy: results from a phase 2 cohort of 101 patients. J Clin Oncol. 2025;43(16 suppl):6504. doi:10.1200/JCO.2025.43.16_suppl.6504.
