FDA Accepts sNDA for INQOVI® Plus Venetoclax for Newly Diagnosed AML Patients Ineligible for Intensive ChemotherapyCombination therapy met primary efficacy endpoint with no new safety signals; would represent the first all-oral regimen for this patient population if approved
Taiho Pharmaceutical Co., Ltd. and its U.S. subsidiary, Taiho Oncology, Inc., announced today that the U.S. Food and Drug Administration (FDA) has accepted their supplemental New Drug Application (sNDA) for INQOVI® (decitabine and cedazuridine) in combination with venetoclax for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) who are not eligible for intensive induction chemotherapy. The application has been granted a standard review, with a Prescription Drug User Fee Act (PDUFA) target action date of February 25, 2026.
The submission is based on data from the Phase 2b ASCERTAIN-V study, which evaluated the safety and efficacy of the INQOVI and venetoclax combination in 101 adult patients with newly diagnosed AML who were deemed unfit for intensive chemotherapy. The combination met its primary endpoint, achieving a complete response (CR) rate of 46.5%. No new safety concerns were identified in the study.
If approved, INQOVI combined with venetoclax would offer the first all-oral treatment option for patients with AML who cannot undergo intensive chemotherapy,” said Harold Keer, MD, PhD, Chief Medical Officer at Taiho Oncology. “This FDA acceptance reflects our continued commitment to delivering innovative treatment options for patients with high unmet medical needs.
Key Findings from the ASCERTAIN-V Study
- Dosing regimen: INQOVI was administered orally on days 1–5 of each 28-day cycle, alongside daily venetoclax.
- Efficacy results:
- CR rate: 46.5% (n=47)
- CR + CR with incomplete hematologic recovery: 63.4% (n=64)
- Median overall survival: 15.5 months
- At 12 months, median duration of response had not been reached; over 75% of patients who achieved CR remained in remission.
- Safety profile:
- No new safety signals reported.
- Grade 3 or higher adverse events occurred in 98% of patients (n=99), most commonly febrile neutropenia (49.5%), anemia (38.6%), and neutropenia (35.6%).
- Early mortality (30-day and 60-day) rates were 3% and 9.9%, respectively.
- No drug-drug interactions were observed between INQOVI and venetoclax.
These data were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and the 2025 European Hematology Association (EHA) Congress.
INQOVI is currently approved in the United States for the treatment of adults with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). If approved for AML, this would mark a significant expansion of its therapeutic use.
An estimated 22,000 individuals in the U.S. will be diagnosed with AML in 2025, and over half are expected to be ineligible for intensive induction chemotherapy, highlighting the urgent need for more accessible and less toxic treatment options.
About INQOVI
This product is an orally administered, fixed dose combination of the anti-cancer DNA hypomethylating agent decitabine, approved in the U.S., together with cedazuridine,5 an inhibitor of cytidine deaminase.6 By inhibiting cytidine deaminase in the gut and the liver, the fixed dose combination is designed to allow for oral delivery of decitabine over five days in a given cycle to achieve comparable systemic exposure to IV decitabine administered over five days.
About Taiho Pharmaceutical Co., Ltd.
Taiho Pharmaceutical, a subsidiary of Otsuka Holdings Co., Ltd. ( https://www.otsuka.com/en/ ), is an R&D-driven specialty pharma focusing on the fields of oncology and immune-related diseases. Its corporate philosophy takes the form of a pledge: “We strive to improve human health and contribute to a society enriched by smiles.” In the field of oncology, in particular, Taiho Pharmaceutical is known as a leading company in Japan for developing innovative medicines for the treatment of cancer, a reputation that is rapidly expanding through their extensive global R&D efforts. In areas other than oncology, as well, the company creates and markets quality products that effectively treat medical conditions and can help improve people’s quality of life. Always putting customers first, Taiho Pharmaceutical also aims to offer consumer healthcare products that support people’s efforts to lead fulfilling and rewarding lives. For more information about Taiho Pharmaceutical, please visit https://www.taiho.co.jp/en/
About Taiho Oncology, Inc.
The mission of Taiho Oncology, Inc. is to improve the lives of patients with cancer, their families and their caregivers. The company specializes in the development and commercialization of orally administered anti-cancer agents for various tumor types. Taiho Oncology has a robust pipeline of small-molecule clinical candidates targeting solid-tumor and hematological malignancies, with additional candidates in pre-clinical development. Taiho Oncology is a subsidiary of Taiho Pharmaceutical Co., Ltd. which is part of Otsuka Holdings Co., Ltd. Taiho Oncology is headquartered in Princeton, New Jersey and oversees its parent company’s European and Canadian operations, which are located in Baar, Switzerland and Oakville, Ontario, Canada.
