The FDA has approved INQOVI (decitabine and cedazuridine) in combination with venetoclax for adults with newly diagnosed acute myeloid leukemia (AML) who are 75 years or older or are ineligible for intensive induction chemotherapy. The decision makes INQOVI plus venetoclax the first and only all-oral combination regimen approved for this patient population, offering an alternative to parenteral hypomethylating agent–based treatments that require frequent clinic visits.
The approval is based on results from the Phase 2 ASCERTAIN-V study in adults with newly diagnosed AML who were not candidates for intensive induction chemotherapy. Efficacy was established using complete remission (CR) and duration of CR (DoCR) as key endpoints. In the trial, 42 patients achieved a CR, for a CR rate of 41.6% (95% CI: 31.9, 51.8), with a median time to CR of two months (range: 0.4 to 15.3 months). The median duration of CR was not reached at the time of analysis (range: 0.5 to 16.3 months).
The prescribing information for INQOVI includes Warnings and Precautions for myelosuppression and embryo-fetal toxicity, and the companies advise clinicians to review the full Important Safety Information and Prescribing Information before use. INQOVI is an orally administered hypomethylating regimen that was previously approved in the U.S. and Canada for the treatment of adults with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML).
“This FDA approval represents a significant milestone for patients with newly diagnosed acute myeloid leukemia who are not candidates for intensive induction chemotherapy,” said Peter Melnyk, president and chief executive officer of Taiho Oncology. He noted that the availability of an all-oral regimen underscores the company’s commitment to advancing innovative, patient-focused therapies in hematologic malignancies.
In 2026, an estimated 22,720 people in the U.S. are expected to be diagnosed with AML, a cancer of the blood and bone marrow, and more than half are likely to be ineligible for intensive induction chemotherapy because of advanced age or other health concerns. Against this backdrop, Taiho executives emphasized the potential of an oral regimen to ease treatment burden.
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