Cedazuridine

Decitabine is a nucleoside metabolic inhibitor that is believed to exert its effects after phosphorylation and direct incorporation into DNA and inhibition of DNA methyltransferase, causing hypomethylation of DNA and cellular differentiation and/or apoptosis. Decitabine inhibits DNA methylation in vitro, which is achieved at concentrations that do not cause major suppression of DNA synthesis.

• INQOVI is a combination of decitabine, a nucleoside metabolic inhibitor, and cedazuridine, a cytidine deaminase inhibitor, 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. ( 1.1 ) In combination with venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.ICD-10: C95.90, D64.9

• None. None.contraindicated

The recommended dosage of INQOVI is 1 tablet (35 mg decitabine and 100 mg cedazuridine) taken orally once daily on Days 1 through 5 of each 28-day cycle. ( 2.2 ) Take INQOVI on an empty stomach. ( 2.2 ) 2.1 Important Administration Information Do NOT substitute INQOVI for an intravenous decitabine product within a cycle. Consider administering antiemetics prior to each dose to minimize nausea and vomiting [see Adverse Reactions (6.1) ]. Take INQOVI on an empty stomach at least 2 hours before or 2 hours after eating. When INQOVI is given in combination with venetoclax, advise patients to take INQOVI 2 hours before or 2 hours after venetoclax. Refer to the venetoclax Prescribing Information for recommended dosage and administration. 2.2 Recommended Dosage INQOVI Monotherapy for MDS or CMML The recommended dosage of INQOVI is 1 tablet (containing 35 mg decitabine and 100 mg cedazuridine) orally once daily on Days 1 through 5 of each 28-day cycle for a minimum of 4 cycles until disease progression or unacceptable toxicity. A complete or partial response may take longer than 4 cycles. INQOVI in Combination with Venetoclax for AML The recommended dosage of INQOVI in combination with venetoclax is 1 tablet (containing 35 mg decitabine and 100 mg cedazuridine) orally once daily on Days 1 through 5 of each 28-day cycle until disease progression or unacceptable toxicity. Match Day 1 of INQOVI dosing with Day 1 of venetoclax dosing for each 28-day cycle. 2.3 Administration Instruct patients of the following: Take INQOVI at approximately the same time each day. Swallow tablets whole.

Myelosuppression : Severe myelosuppression, including fatal adverse reactions and infectious complications can occur. Obtain complete blood cell counts prior to initiation of INQOVI, prior to each cycle, and as clinically indicated to monitor for response and toxicity. Delay the next cycle and resume at the same or reduced dose as recommended. ( 2.3 , 5.1 ) Embryo-Fetal Toxicity : Can cause fetal harm. Advise patients of reproductive potential of the potential risk to a fetus and to use effective contraception. ( 5.2 , 8.1 , 8.3 ) 5.1 Myelosuppression INQOVI 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.

The following clinically significant adverse reactions are described elsewhere in the labeling: Myelosuppression [see Warnings and Precautions (5.1) ] In MDS or CMML, the most common adverse reactions (incidence ≥ 20%) are fatigue, constipation, hemorrhage, myalgia, mucositis, arthralgia, nausea, dyspnea, diarrhea, rash, dizziness, febrile neutropenia, edema, headache, cough, decreased appetite, upper respiratory tract infection, pneumonia, and transaminase increased. The most common Grade 3 or 4 laboratory abnormalities (≥ 50%) were leukocytes decreased, platelet count decreased, neutrophil count decreased, and hemoglobin decreased. ( 6.1 ) In AML in combination with venetoclax, the most common adverse reactions (incidence ≥ 20%) are neutropenia, febrile neutropenia, thrombocytopenia, hemorrhage, anemia, infection (bacterial/viral), diarrhea, fatigue, mucositis, constipation, arthralgia, dyspnea, decreased appetite, edema, nausea, white blood cell count decreased, sepsis, pneumonia, rash, transaminitis, myalgia, arrhythmia, and abdominal pain. The most common Grade 3 or 4 laboratory abnormalities (≥ 50%) were leukocytes decreased, lymphocytes decreased, platelets decreased, and hemoglobin decreased. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Taiho Oncology, Inc. at 1-844-878-2446 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .

Lactation : Advise not to breastfeed. ( 8.2 ) Infertility : Can impair fertility. ( 8.3 ) 8.1 Pregnancy Risk Summary Based on findings from human data, animal studies, and its mechanism of action [see Clinical Pharmacology (12.1) ] , INQOVI can cause fetal harm when administered to a pregnant woman. A single published case report of intravenous decitabine use throughout the first trimester during pregnancy describes adverse developmental outcomes, including major birth defects (structural abnormalities). In animal reproduction studies, intravenous administration of decitabine to pregnant mice and rats during organogenesis at doses approximately 7% of the recommended human dose on a body surface area (mg/m 2 ) basis caused adverse developmental outcomes, including increased embryo-fetal mortality, alterations to growth, and structural abnormalities (see Data ) . Advise pregnant women of the potential risk to a fetus. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Human Data There are no available data on INQOVI use in pregnant women.

Metabolism

The pharmacokinetics of decitabine and cedazuridine were characterized following administration of INQOVI in patients with MDS / CMML and AML are shown in Table 7 . Approximately dose-proportional increases in peak concentrations (C max ) and AUC over the dosing interval were observed for decitabine following administration of oral decitabine at 20 mg to 40 mg once daily (0.6 to 1.1 times the recommended dose) in combination with 100 mg oral cedazuridine, and for cedazuridine following administration of oral cedazuridine at 40 to 100 mg once daily (0.4 to 1.0 times the recommended dose) in com…

• Jul 7, 2020NDANDA212576Taiho Oncology

1. 1Death10013%
2. 2Inappropriate Schedule Of Product Administration9512%
3. 3Fatigue9212%
4. 4Platelet Count Decreased9112%
5. 5Product Use In Unapproved Indication8011%
6. 6Cytopenia749.7%
7. 7White Blood Cell Count Decreased699.1%
8. 8Transfusion587.6%
9. 9Off Label Use577.5%
10. 10Nausea506.6%
11. 11Asthenia435.7%
12. 12Haemoglobin Decreased435.7%
13. 13Pancytopenia395.1%
14. 14Neutropenia385.0%
15. 15Febrile Neutropenia364.7%

The 10 most recently updated of 78 ClinicalTrials.gov registrations naming Cedazuridine as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia
  Recruiting · Phase 1 · Interventional · 47 enrolled · National Cancer Institute (NCI)
  NCT07012044updated 2026-06-12
• Testing the Addition of an Anti-cancer Drug, ASTX727 (Cedazuridine, Decitabine), to Chemotherapy (Paclitaxel) and Immunotherapy (Pembrolizumab) for Metastatic Triple-Negative Breast Cancer
  Recruiting · Phase 1 · Interventional · 32 enrolled · National Cancer Institute (NCI)
  NCT05673200updated 2026-06-11
• A Phase Ia/Ib Open-label, Multiple Dose, Study to Determine the Recommended Dose, Evaluate PKs, PDs, Safety, and Activity of Venetoclax in Combination With Oral Decitabine/Cedazuridine (ASTX727) in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML)
  Recruiting · Phase 1 · Interventional · 40 enrolled · M.D. Anderson Cancer Center
  NCT06191978updated 2026-06-11
• Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)
  Recruiting · Phase 2 · Interventional · 54 enrolled · National Cancer Institute (NCI)
  NCT06577441updated 2026-06-11
• MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
  Recruiting · Phase 2 · Interventional · 2,000 enrolled · National Cancer Institute (NCI)
  NCT05564390updated 2026-06-11
• Venetoclax in Combination With ASTX727 for the Treatment of Treatment-Naive High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
  Active not recruiting · Phase 1 · Phase 2 · Interventional · 52 enrolled · M.D. Anderson Cancer Center
  NCT04655755updated 2026-06-11
• ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
  Recruiting · Phase 1 · Phase 2 · Interventional · 42 enrolled · M.D. Anderson Cancer Center
  NCT05010122updated 2026-06-11
• Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes
  Recruiting · Phase 1 · Interventional · 54 enrolled · National Cancer Institute (NCI)
  NCT06484062updated 2026-06-11
• Comparing New Treatments for People With Newly Diagnosed Acute Myeloid Leukemia That Has an IDH2 Gene Change (A MyeloMATCH Treatment Trial)
  Recruiting · Phase 2 · Interventional · 93 enrolled · National Cancer Institute (NCI)
  NCT06672146updated 2026-06-11
• Venetoclax and ASTX727 for the Treatment of Relapsed, Refractory, or Newly Diagnosed Acute Myeloid Leukemia
  Active not recruiting · Phase 2 · Interventional · 85 enrolled · M.D. Anderson Cancer Center
  NCT04746235updated 2026-06-11

Frequently asked questions

How does Cedazuridine work?

Decitabine is a nucleoside metabolic inhibitor that is believed to exert its effects after phosphorylation and direct incorporation into DNA and inhibition of DNA methyltransferase, causing hypomethylation of DNA and cellular differentiation and/or apoptosis. Decitabine inhibits DNA methylation in vitro, which is achieved at concentrations that do not cause major suppression of DNA synthesis.

What is Cedazuridine used for?

According to FDA labeling, Cedazuridine carries indications including: INQOVI is a combination of decitabine, a nucleoside metabolic inhibitor, and cedazuridine, a cytidine deaminase inhibitor, 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. ( 1.1 ) In combination with venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What are the brand names for Cedazuridine?

Cedazuridine is marketed under brand names including Inqovi.

What are the contraindications for Cedazuridine?

Cedazuridine labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.