Oral Hypomethylating Regimen Under FDA Review for Newly Diagnosed AML

The FDA has accepted a Supplemental New Drug Application (sNDA) for decitabine and cedazuridine (INQOVI) plus venetoclax for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy. The FDA is expected to make a decision by February 25, 2026.¹

The sNDA is supported by the phase 2b portion of the ASCERTAIN-V trial presented at the European Hematology Association 2025 Congress. Among 101 adult patients who received decitabine-cedazuridine plus venetoclax, 46.5% (95% confidence interval [CI], 36.5–56.7%) achieved complete remission (CR), and 63.4% (95% CI, 53.2–72.7%) achieved CR/CR with incomplete hematologic recovery. The median time to CR was 2.4 months.²

In terms of safety, 98.0% of patients experienced grade 3 or higher adverse events, most commonly febrile neutropenia (49.5%), anemia (38.6%), and neutropenia (35.6%). The 30-day mortality rate was 3.0%, and the 60-day mortality rate was 9.9%.²

Decitabine-cedazuridine is currently indicated in the US to treat adult patients with myelodysplastic syndromes and chronic myelomonocytic leukemia.¹

“We have an unwavering dedication to developing innovative new cancer treatments, and the FDA’s acceptance of our sNDA for INQOVI in combination with venetoclax highlights the need for novel approaches in AML,” said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology, in a press release.¹ “If approved for patients with AML who are not eligible to undergo intensive induction chemotherapy, INQOVI in combination with venetoclax would offer the first all-oral alternative to current therapies.”