FDA Accepts Taiho's Application for INQOVI-Venetoclax Combination in Acute Myeloid Leukemia
Key Insights
The FDA has accepted Taiho Oncology (search)'s supplemental new drug application for INQOVI (decitabine and cedazuridine) plus venetoclax to treat adults with newly diagnosed acute myeloid leukemia who are ineligible for intensive induction chemotherapy.
The Phase 2b ASCERTAIN-V trial demonstrated a 46.5% complete response rate in 101 patients, with median overall survival estimated at 15.5 months and no new safety concerns reported.
If approved, this would represent the first all-oral combination treatment option for AML patients ineligible for intensive chemotherapy, with a PDUFA target action date of February 25, 2026.
The U.S. Food and Drug Administration has accepted Taiho Oncology (search)'s supplemental new drug application for INQOVI (decitabine and cedazuridine) in combination with venetoclax as a treatment for adults with newly diagnosed acute myeloid leukemia who are ineligible for intensive induction chemotherapy. The FDA assigned a standard review with a Prescription Drug User Fee Act target action date of February 25, 2026.
The application is supported by results from ASCERTAIN-V, a Phase 2b study that enrolled 101 adult patients with newly diagnosed AML who were deemed ineligible for intensive induction chemotherapy. If approved, this combination would represent the first all-oral treatment alternative for this patient population.
Clinical Trial Results Demonstrate Efficacy
The ASCERTAIN-V trial met its primary endpoint with a complete response rate of 46.5% (n=47). In the 28-day treatment cycles, patients received INQOVI on days one through five and venetoclax daily, with a median follow-up period of 11.2 months.
Secondary endpoint analysis revealed that complete response plus complete response with incomplete hematologic recovery totaled 63.4% (n=64). The median overall survival was estimated to be 15.5 months. At 12 months, the median duration of response had not been reached, and over 75% of patients achieving complete response status remained in complete response.
"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 at Taiho Oncology (search). "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."
Safety Profile Remains Manageable
No new safety concerns were reported in the study. Adverse events of grade 3 and higher were reported in 98% of patients (n=99), with the most commonly reported being febrile neutropenia (49.5%), anemia (38.6%), and neutropenia (35.6%). No drug-drug interactions were observed between INQOVI and venetoclax.
At 30 and 60 days after the first dose of INQOVI, deaths attributed to either adverse events or disease progression totaled 3% (n=3) and 9.9% (n=10) of study participants, respectively.
Addressing Significant Unmet Medical Need
Approximately 22,000 people in the U.S. will receive a diagnosis of AML, a cancer of the blood and bone marrow, in 2025. More than half of those patients will likely be deemed ineligible for intensive induction chemotherapy, highlighting the significant unmet medical need for this patient population.
INQOVI is currently the first and only oral hypomethylating agent approved by the FDA for the treatment of adults with intermediate and high-risk myelodysplastic syndromes and chronic myelomonocytic leukemia. The drug is an orally administered, fixed-dose combination of decitabine and cedazuridine, an inhibitor of cytidine deaminase. By inhibiting cytidine deaminase in the gut and liver, the combination is designed to allow for oral delivery of decitabine over five days in a given cycle to achieve comparable systemic exposure to intravenous decitabine administered over five days.
The ASCERTAIN-V study results were presented at the 2025 American Society of Clinical Oncology Annual Meeting and the 2025 European Hematology Association Congress.