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News|Articles|May 13, 2026

FDA Approves First All-Oral Regimen for AML

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Key Takeaways

  • Regulatory clearance creates an at-home, 28-day cycle option using oral decitabine/cedazuridine on days 1–5 plus venetoclax 400 mg daily after a ramp-up cycle.
  • Efficacy signals in ASCERTAIN-V included 46.5% CR and 63.4% CR/CRi, with median OS 15.5 months and durable remissions beyond 12 months.
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Taiho submitted the supplemental new drug application in April 2025 for decitabine/cedazuridine and venetoclax, and the FDA accepted it for review in July 2025.

The FDA has approved a novel combination treatment for newly diagnosed acute myeloid leukemia (AML) for patients not eligible to undergo intensive induction chemotherapy who are 75 years and older. Decitabine and cedazuridine (Inqovi; Taiho), formerly the investigative AStx727-07, plus venetoclax, is now the first all-oral combination therapy for this indication.1,2

Data from the phase 2 ASCERTAIN-V trial (NCT04657081) supported Taiho’s application and the FDA’s decision. These data show the investigation met its complete response (CR) end points of interest and that no new safety concerns were reported. A total of 101 patients were included in the trial.

The overall CR rate was 41.6% (95% CI, 31.9%-51.8%), median time to CR was 2 months (range, 0.4-15.3), and median duration of CR was not reached (range, 0.5-16.3 months).1 CR plus CR with incomplete hematologic recovery rate was 63.4% (n = 64), a key secondary end point.2 Median estimated overall survival (OS) was 15.5 months. By the 12-month mark, median duration of response (DOR) had not been reached, and more than 75% of patients who had already achieved a CR remained at that status.

Ninety-eight percent of the trial population reported grade 3 or higher adverse events (AEs), with the top 3 being febrile neutropenia (49.5%), anemia (38.6%), and neutropenia (35.6%). There were 13 deaths overall, 3 because of AEs and 10 from disease progression; this outcome was evaluated at 30 and 60 days after the final dose of decitabine and cedazuridine.

The ongoing international ASCERTAIN-V trial—the estimated completion date is December 31, 2026—required patients to be 18 years or older, have newly diagnosed AML per World Health Organization 2016 criteria, have a life expectancy of at least 3 months, be ineligible for intensive induction chemotherapy (eg, older age or comorbidities), have an Eastern Cooperative Oncology Group Performance Status of 0 to 3, not be pregnant or breastfeeding, agree to use contraception for the study’s duration and 3 months after the final dose and to not donate sperm, and be able to give legal informed consent.3 As with the phase 1 portion, the phase 2 portion was a single-arm, open-label, multicenter, nonrandomized interventional investigation. Treatment cycles were 28 days. Decitabine and cedazuridine were administered on days 1 through 5, and venetoclax at 400 mg was given daily3—all after 1 cycle of venetoclax ramp-up dosing.4 Median follow-up was 11.2 months.

Overall, the primary outcomes of interest were venetoclax area under the curve from time 0 to 24 hours (AUC0-24) with/without AStx727-07 (phase 1 and phase 2A), venetoclax maximum observed concentration (Cmax) with/without AStx727-07 (phase 1 and phase 2A), and CR (phase 2A and phase 2B).3 There were 13 secondary outcomes of interest, including AUC0-24 for decitabine and cedazuridine, Cmax for decitabine and cedazuridine, treatment-emergent AEs, DOR, and OS.

Decitabine and cedazuridine is also currently approved to treat myelodysplastic syndromes and chronic myelomonocytic leukemia in adult patients,5 and venetoclax is currently approved to treat chronic lymphocytic leukemia or small lymphocytic lymphoma.4

References

  1. FDA approves oral combination of decitabine and cedazuridine tablets with venetoclax for newly diagnosed acute myeloid leukemia. FDA. May 13, 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
  2. Taiho Oncology and Taiho Pharmaceutical announce U.S. FDA acceptance of supplemental new drug application for Inqovi in combination with venetoclax to treat patients with acute myeloid leukemia. News release. Taiho Oncology. July 9, 2025. Accessed May 13, 2026. https://www.taihooncology.com/us/news/taiho-oncology-and-taiho-pharmaceutical-announce-us-fda-acceptance-of-supplemental-new-drug-application-for-inqovi-in-combination-with-venetoclax-to-treat-patients-with-acute-myeloid-leukemia/
  3. Pharmacokinetics, safety, and efficacy of ASTX727 in combination with venetoclax in acute myeloid leukemia (AML). ClinicalTrials.gov. Updated December 8, 2025. Accessed May 13, 2026. https://clinicaltrials.gov/study/NCT04657081
  4. Combination of decitabine-cedazuridine plus venetoclax would be all-oral regimen for patients with ND AML. ASH Clinical News. February 2026. Accessed May 13, 2026. https://ashpublications.org/ashclinicalnews/news/9132/combination-of-decitabine-cedazuridine-plus
  5. Joszt L. FDA approves at-home therapy to treat myelodysplastic syndromes. AJMC®. July 11, 2020. Accessed May 13, 2026. https://www.ajmc.com/view/fda-approves-athome-therapy-to-treat-myelodysplastic-syndromes

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