FDA Approves First All-Oral AML Regimen as Inqovi Plus Venetoclax Clears for Patients Ineligible for Intensive Chemotherapy

Overview

The U.S. Food and Drug Administration on May 13, 2026 approved INQOVI (decitabine and cedazuridine) in combination with venetoclax for the treatment of adults with newly diagnosed acute myeloid leukemia who are 75 years or older, or who have comorbidities that preclude the use of intensive induction chemotherapy, Taiho Oncology announced via BioSpace. The approval marks the first and only all-oral combination treatment regimen available for this patient population, replacing parenteral hypomethylating agent-based regimens that require frequent clinic visits.

AML is estimated to account for approximately 22,720 new diagnoses in the United States in 2026, with a median age at diagnosis of 70 years, according to the National Cancer Institute’s SEER program. The disease is most frequently diagnosed among people aged 65–74, who account for 26.6% of new cases, with the 75–84 age group comprising an additional 24.9%.

What We Know

The approval was supported by the single-arm, open-label Phase 2 ASCERTAIN-V trial (NCT04657081), in which 101 patients with newly diagnosed AML ineligible for intensive chemotherapy received the oral combination, AML Hub reported. The primary efficacy endpoint — complete remission rate — was 41.6% (95% CI, 31.9–51.8), with 42 of 101 patients achieving complete remission, per the Colorado Association of Health Plans. Median time to complete remission was 2 months (range: 0.4–15.3 months), and median duration of complete remission was not reached (range: 0.5–16.3 months).

INQOVI is a fixed-dose combination tablet that pairs decitabine, a DNA hypomethylating agent, with cedazuridine, a cytidine deaminase inhibitor, enabling oral delivery that achieves comparable systemic exposure to intravenous decitabine, BioSpace reported. Venetoclax, the BCL-2 inhibitor widely used in AML, was already approved for this indication in combination with injectable azacitidine or subcutaneous decitabine. The new regimen replaces the injection-based hypomethylating agent component with an oral tablet, Pharmaceutical Technology noted.

The standard injectable hypomethylating agent azacitidine is administered subcutaneously or intravenously on days 1 through 7 of each treatment cycle, a schedule that translates to repeated clinic visits each month, per a 2026 treatment algorithm review in Blood Cancer Journal. That same review notes that venetoclax-based hypomethylating agent combinations are not time-limited therapies and are frequently accompanied by significant myelosuppression.

Taiho Oncology’s chief medical officer, Harold Keer, MD, PhD, said in the company’s announcement that “this approval marks an important step forward in expanding how treatment can be delivered for this patient population, offering an all-oral option that can potentially reduce the overall treatment burden associated with receiving treatment in hospitals or infusion centers,” per PharmExec. Peter Melnyk, president and chief executive officer of Taiho Oncology, said “with the approval of an all-oral regimen, Inqovi in combination with venetoclax brings a new treatment option to this patient population and underscores our commitment to advancing innovative, patient-focused therapies in hematologic malignancies,” per PharmExec.

Safety profile. In the ASCERTAIN-V trial, serious adverse reactions occurred in 82% of patients, BioSpace reported. The most common Grade 3 or 4 toxicities were thrombocytopenia (69%), anemia (50%), febrile neutropenia (52%), and neutropenia (48%). Fatal adverse reactions occurred in 8% of patients, with sepsis accounting for 5%, and dyspnea, myocardial infarction, hemolytic anemia, and tumor lysis syndrome each accounting for 1%.

What We Don’t Know

• Comparative efficacy data. The ASCERTAIN-V trial was a single-arm study without a direct comparator arm. While the current standard of care — injectable azacitidine in combination with venetoclax — demonstrated a complete remission rate of approximately 36% in the pivotal VIALE-A trial, no head-to-head comparison between the oral and injectable regimens has been published.

• Long-term survival outcomes. Because median duration of complete remission was not reached in ASCERTAIN-V at the time of the FDA review, the impact on overall survival remains to be established in longer follow-up.

• Pricing and access. Taiho Oncology has not yet announced U.S. list pricing for the new indication.

Analysis

The approval completes a shift in AML frontline therapy that began when azacitidine plus venetoclax first demonstrated survival benefit over azacitidine alone in the VIALE-A trial. Where earlier iterations of low-intensity AML treatment still required patients to travel to infusion centers each month for injections, an all-oral regimen removes that logistical barrier — a meaningful quality-of-life consideration for older and more frail patients.

Over half of newly diagnosed AML patients are considered ineligible for intensive chemotherapy due to age and comorbidities, Pharmaceutical Technology reported, making the addressable population for low-intensity oral combinations substantial. Inqovi had already received FDA approval for myelodysplastic syndromes in July 2020, giving physicians and pharmacies familiarity with the tablet formulation before its AML combination use.

The five-year survival rate for AML stands at 33.4% across all ages, per SEER, but outcomes are considerably worse in older populations. For a disease whose median patient is 70 years old at diagnosis, reducing the logistical burden of treatment — without sacrificing efficacy — has long been identified as a meaningful clinical goal. Whether the oral regimen can demonstrate improved overall survival in longer follow-up, and whether community oncologists adopt it at the same rate as the injectable standard, will shape its real-world impact.