FDA OKs Oral Decitabine/Cedazuridine Plus Venetoclax in Newly Diagnosed AML

The FDA approved oral decitabine and cedazuridine tablets (Inqovi) plus venetoclax (Venclexta) for patients 75 years and older with newly diagnosed acute myeloid leukemia (AML), according to a news release from the agency.¹ The regimen is also indicated for patients with comorbidities that make them ineligible for intensive induction chemotherapy.

Supporting data for the approval came from the phase 1/2 ASTX727-07 study (NCT04657081), in which investigators evaluated the regimen among 101 evaluable patients. The treatment regimen produced complete responses (CRs) in 41.6% (n = 42; 95% CI, 31.9%-51.8%) of patients. Additionally, the median time to CR was 2 months (range, 0.4-15.3), and the median duration of CR was not reached (range, 0.5-16.3).

The regimen’s prescribing information includes warnings and precautions for embryo-fetal toxicity and myelosuppression. The FDA approved the tablets at a dosage of 35 mg of decitabine and 100 mg of cedazuridine taken orally once daily on days 1 to 5 of each 28-day cycle until progressive disease or unacceptable toxicity.

The trial’s primary end points included the CR rate and pharmacokinetics.² Secondary end points included time to response, duration of response, and overall survival.

References

1. FDA approves oral combination of decitabine and cedazuridine tablets with venetoclax for newly diagnosed acute myeloid leukemia. News release. FDA. May 13, 2026. Accessed May 13, 2026. https://tinyurl.com/ykk7yhfz
2. 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://tinyurl.com/4u35pzjd