Taiho Oncology announced FDA approval of INQOVI plus venetoclax as the first all-oral regimen for newly diagnosed AML patients aged 75+ or ineligible for intensive chemotherapy.
Written By: Sana Khan, BPharm
Reviewed By: Pharmacally Editorial Team
Taiho Pharmaceutical Co., Ltd., part of the Otsuka Group, and its U.S. subsidiary Taiho Oncology, Inc., announced that the U.S. Food and Drug Administration has approved INQOVI® (decitabine and cedazuridine) in combination with venetoclax for adults with newly diagnosed acute myeloid leukemia (AML) who are aged 75 years or older or who are not eligible for intensive induction chemotherapy. The approval was granted through a supplemental New Drug Application (sNDA), expanding INQOVI’s U.S. label into frontline AML.
According to the company, the regimen is the first FDA-approved all-oral treatment combination for this patient population, providing an alternative to injectable hypomethylating agent (HMA)-based regimens that often require frequent hospital or clinic visits. Injectable HMAs such as azacitidine and decitabine, used in combination with venetoclax, are current standards in this setting, making the approval a notable shift toward an oral therapy option.
The FDA decision was supported by data from the Phase 2 ASCERTAIN-V trial (NCT04657081), which evaluated INQOVI plus venetoclax in adults with newly diagnosed AML who were ineligible for intensive induction chemotherapy. Efficacy was assessed based on complete remission (CR) and duration of complete remission (DoCR).
In the study, 42 patients achieved complete remission, corresponding to a CR rate of 41.6% (95% CI: 31.9–51.8). The median time to complete remission was two months, with responses observed between 0.4 and 15.3 months, and the median duration of complete remission was not reached at the time of analysis, with observed durations ranging from 0.5 to 16.3 months.
The U.S. prescribing information for the regimen includes warnings and precautions related to myelosuppression and embryo-fetal toxicity. The oral administration of the regimen may offer greater convenience for appropriate patients and could help reduce reliance on infusion centers, although longer-term outcomes such as survival and quality of life will require continued evaluation.
INQOVI was previously approved in the United States and Canada for adults with myelodysplastic syndromes and chronic myelomonocytic leukemia. The oral fixed-dose combination includes decitabine, a DNA hypomethylating agent, and cedazuridine, a cytidine deaminase inhibitor designed to enable oral delivery of decitabine with systemic exposure comparable to intravenous administration.
Peter Melnyk, President and Chief Executive Officer of Taiho Oncology, said the approval marks an important milestone for patients with newly diagnosed AML who cannot undergo intensive induction chemotherapy. He added that the all-oral regimen expands treatment options for patients with hematologic malignancies.
Harold Keer, MD, PhD, Chief Medical Officer of Taiho Oncology, said the approval supports broader use of oral anti-cancer regimens and may help reduce the treatment burden associated with hospital or infusion-center visits for eligible patients. The approval also positions Taiho more directly against established injectable HMA-plus-venetoclax regimens, including azacitidine plus venetoclax, in the frontline AML setting.
According to company-provided estimates, around 22,720 people in the United States are expected to be diagnosed with AML in 2026, and more than half may be ineligible for intensive induction chemotherapy because of age or other health-related factors.
Reference
About the Writer
Sana Jamil Khan (LinkedIn) is a B. Pharm graduate with a strong interest in medical writing and scientific communication. Her work focuses on interpreting clinical research, exploring developments in pharmaceutical science, and presenting complex medical information in a clear and accessible manner. She is particularly interested in topics related to human clinical studies, drug safety observations, and emerging therapeutic research.