Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd., today announced that the U.S. Food and Drug Administration (FDA) has approved INQOVI (decitabine and cedazuridine) plus venetoclax as a treatment for adults with newly diagnosed acute myeloid leukemia (AML) who are 75 years or older or who are ineligible for intensive induction chemotherapy. INQOVI in combination with venetoclax is the first and only all-oral combination treatment regimen approved for this patient population, offering an alternative to parenteral hypomethylating agent–based regimens that require frequent clinic visits.
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The approval was supported by results from the Phase 2 ASCERTAIN-V study of INQOVI plus venetoclax in adult patients with newly diagnosed AML who were ineligible for intensive induction chemotherapy.1
Efficacy was established based on complete remission (CR) and the duration of CR (DoCR). Duration of remission was defined as the time from first CR until disease relapse or death from any cause, whichever occurred first. In combination with venetoclax, 42 patients achieved a CR (41.6%, 95% CI: 31.9, 51.8) with a median time to CR of two months (range: 0.4 to 15.3 months). The median duration of CR was not reached (range: 0.5 to 16.3 months).
The Prescribing Information contains Warnings and Precautions for myelosuppression and embryo-fetal toxicity. Please see the Important Safety Information and Prescribing Information for more details.
INQOVI is an orally administered hypomethylating regimen previously approved in the U.S. and Canada for the treatment of adults with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML).2
“This FDA approval represents a significant milestone for patients with newly diagnosed acute myeloid leukemia who are not candidates for intensive induction chemotherapy,” said Peter Melnyk, President and Chief Executive Officer of Taiho Oncology. “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.”
In 2026, an estimated 22,720 people in the U.S. will be diagnosed with AML, a cancer of the blood and bone marrow.3 More than half of those patients are likely to be ineligible for intensive induction chemotherapy due to advanced age or health concerns.4
“As a leader in the development of oral anti-cancer regimens, we are proud that INQOVI in combination with venetoclax will now be available for newly diagnosed patients with acute myeloid leukemia in the U.S.,” said Harold Keer, MD, PhD, Chief Medical Officer of Taiho Oncology. “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. We believe this approach has the potential to make a meaningful impact for patients and caregivers.”
*ASCERTAIN-V Study: AStx727-07: decitabine + CEdazuRidine TreAtment IN AML, adding Venetoclax
About INQOVI
INQOVI is an orally administered, fixed-dose combination of the DNA hypomethylating agent decitabine 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 the oral delivery of decitabine to achieve comparable systemic exposure to that of IV decitabine.
Indications and Important Safety Information
INDICATIONS
INQOVI is indicated for treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.
INQOVI is indicated in combination with venetoclax for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) who are 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Myelosuppression
INQOVI as Monotherapy for MDS or CMML
In patients with MDS or CMML, INQOVI can cause severe myelosuppression, including fatal adverse reactions. Based on laboratory values, new or worsening thrombocytopenia occurred in 82% of patients, with Grade 3 or 4 occurring in 76%. Neutropenia occurred in 73% of patients, with Grade 3 or 4 occurring in 71%. Anemia occurred in 71% of patients, with Grade 3 or 4 occurring in 55%. Febrile neutropenia occurred in 33% of patients, with Grade 3 or 4 occurring in 32%. Thrombocytopenia, neutropenia, anemia, and febrile neutropenia are the most frequent cause of INQOVI dose reduction or interruption, occurring in 36% of patients. Permanent discontinuation due to myelosuppression (febrile neutropenia) occurred in 1% of patients. Myelosuppression and worsening neutropenia may occur more frequently in the first or second treatment cycles and may not necessarily indicate progression of underlying MDS.
Fatal and serious infectious complications can occur with INQOVI. Pneumonia occurred in 21% of patients, with Grade 3 or 4 occurring in 15%. Sepsis occurred in 14% of patients, with Grade 3 or 4 occurring in 11%. Fatal pneumonia occurred in 1% of patients, fatal sepsis in 1%, and fatal septic shock in 1%.
Obtain complete blood cell counts prior to initiation of INQOVI, prior to each cycle, and as clinically indicated to monitor response and toxicity. Administer growth factors and anti‑infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose as recommended.
INQOVI in Combination with Venetoclax for AML
In patients with AML, INQOVI can cause severe myelosuppression, including fatal adverse reactions, when given in combination with venetoclax. Based on laboratory values in Study ASTX727-07 Phase 2 new or worsening thrombocytopenia occurred in 70% of patients, with Grade 3 or 4 occurring in 69%. Neutropenia occurred in 48% of patients, with Grade 3 or 4 occurring in 48%. Anemia occurred in 54% of patients, with Grade 3 or 4 occurring in 50%. Febrile neutropenia occurred in 52% of patients, with Grade 3 or 4 occurring in 52%. Thrombocytopenia, neutropenia, anemia, and febrile neutropenia were a frequent cause of INQOVI and/or venetoclax dose reduction or interruption. Dose reductions of INQOVI due to neutropenia and thrombocytopenia occurred in 4% and 1% of patients, respectively. Dose interruptions of INQOVI due to neutropenia, febrile neutropenia, thrombocytopenia, and anemia occurred in 40%, 11%, 8%, and 2% of patients, respectively.
Fatal and serious infectious complications can occur during treatment with INQOVI and venetoclax. Pneumonia occurred in 25% of patients, with Grade 3 or 4 occurring in 20%. Sepsis occurred in 28% of patients, with Grade 3 or 4 occurring in 18%. Fatal pneumonia occurred in 2% of patients and fatal sepsis in 8%.
Obtain complete blood cell counts prior to initiation of INQOVI with venetoclax, prior to each cycle, and as clinically indicated to monitor response and toxicity. Administer growth factors and anti-infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose as recommended.
Embryo-Fetal Toxicity
Advise patients of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with INQOVI and for 6 months after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with INQOVI and for 3 months after the last dose.
ADVERSE REACTIONS
INQOVI as Monotherapy for MDS or CMML
Serious adverse reactions occurred in 68% of patients who received INQOVI. Serious adverse reactions in > 5% of patients included febrile neutropenia (30%), pneumonia (14%), and sepsis (13%). Fatal adverse reactions occurred in 6% of patients. These included sepsis (1%), septic shock (1%), pneumonia (1%), respiratory failure (1%), and one case each of cerebral hemorrhage and sudden death.
The most common adverse reactions (≥ 20%) were fatigue (55%), constipation (44%), hemorrhage (43%), myalgia (42%), mucositis (41%), arthralgia (40%), nausea (40%), dyspnea (38%), diarrhea (37%), rash (33%), dizziness (33%), febrile neutropenia (33%), edema (30%), headache (30%), cough (28%), decreased appetite (24%), upper respiratory tract infection (23%), pneumonia (21%), and transaminase increased (21%). The most common Grade 3 or 4 laboratory abnormalities (≥ 50%) were leukocytes decreased (81%), platelet count decreased (76%), neutrophil count decreased (71%), and hemoglobin decreased (55%).
INQOVI in Combination with Venetoclax for AML
Serious adverse reactions occurred in 82% of patients who received INQOVI+VEN. Serious adverse reactions in > 5% of patients included febrile neutropenia (31%), sepsis (22%), pneumonia (15%), infection (bacterial/viral) (10%), hemorrhage (9%), and dyspnea (6%). Fatal adverse reactions occurred in 8% of patients who received INQOVI+VEN. These included sepsis (5%), dyspnea (2%), myocardial infarction (1%), hemolytic anemia (1%), and tumor lysis syndrome (1%).
The most common adverse reactions (≥ 20%) were neutropenia (60%), febrile neutropenia (52%), thrombocytopenia (52%), hemorrhage (42%), anemia (41%), infection (bacterial/viral) (40%), diarrhea (38%), fatigue (36%), mucositis (36%), constipation (36%), arthralgia (35%), decreased appetite (31%), edema (31%), nausea (31%), dyspnea (30%), white blood cell count decreased (28%), sepsis (28%), pneumonia (25%), rash (25%), transaminitis (24%), myalgia (23%), arrhythmia (21%), and abdominal pain (21%). The most common Grade 3 or 4 laboratory abnormalities (≥ 20%) were decreased leukocytes (91%), decreased lymphocytes (81%), decreased platelets (69%), decreased hemoglobin (50%), and decreased neutrophils (48%).
USE IN SPECIFIC POPULATIONS
Lactation
Because of the potential for serious adverse reactions in the breastfed child, advise women not to breastfeed during treatment with INQOVI and for 2 weeks after the last dose.
Renal Impairment
No dosage modification of INQOVI is recommended for patients with mild or moderate renal impairment (creatinine clearance [CLcr] of 30 to 89 mL/min based on Cockcroft-Gault). Due to the potential for increased adverse reactions, monitor patients with moderate renal impairment (CLcr 30 to 59 mL/min) frequently for adverse reactions. INQOVI has not been studied in patients with severe renal impairment (CLcr 15 to 29 mL/min) or end-stage renal disease (ESRD: CLcr <15 mL/min).
Please see full Prescribing Information.
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.
For more information, visit https://www.taihooncology.com/, and follow us on LinkedIn and X.
Taiho Oncology and the Taiho Oncology logo are registered trademarks of Taiho Pharmaceutical Co., Ltd.
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/.
References
- Zeidan A, Griffiths E, Dinardo C, et al. An all-oral regimen of decitabine-cedazuridine (DEC-C) plus venetoclax (VEN) in patients (pts) with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive induction chemotherapy: Results from a phase 2 cohort of 101 pts. Presented at: the 2025 Annual Meeting of the American Society of Clinical Oncology; May 30-June 3, 2025; Chicago. Abstract 6504. https://ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.6504.
- Oncology Practice Management. FDA Approves Inqovi, a New Oral Combination Therapy, for Patients with MDS. Published July 7, 2020. Accessed October 10, 2025. https://oncpracticemanagement.com/issues/2020/august-2020-vol-10-no-8/1719-fda-approves-inqovi-a-new-oral-combination-therapy-for-patients-with-mds.
- American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Revised January 13, 2026. Accessed March 27, 2026. https://www.cancer.org/cancer/types/acute-myeloid-leukemia/about/key-statistics.html.
- Heuser M, Fernandez C, Hauch O, Klibanov OM, Chaudhary T, Rives V. Therapies for acute myeloid leukemia in patients ineligible for standard induction chemotherapy: a systematic review. Future Oncol. 2022;19(11):789-810. https://doi.org/10.2217/fon-2022-1286.
- Oganesian A, Redkar S, Taverna P, Choy G, Joshi-Hangal R, Azab M. Preclinical data in cynomolgus (cyn) monkeys of ASTX727, a novel oral hypomethylating agent (HMA) composed of low-dose oral decitabine combined with a novel cytidine deaminase inhibitor (CDAi) E7727 [ASH Abstract]. Blood 2013;122(21): Abstract 2526. https://doi.org/10.1182/blood.V122.21.2526.2526.
- Ferraris D, Duvall B, Delahanty G, et al. Design, synthesis, and pharmacological evaluation of fluorinated tetrahydrouridine derivatives as inhibitors of cytidine deaminase. J Med Chem. 2014; 57:2582-2588. DOI: 10.1021/jm401856k.
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