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Decitabine/Cedazuridine
Decitabine/Cedazuridine is a DNA methyltransferase inhibitor; hypomethylating agent Small molecule drug developed by Astex Pharmaceuticals, Inc.. It is currently in Phase 3 development for Myelodysplastic syndromes (MDS), Acute myeloid leukemia (AML). Also known as: ASTX727, INQOVI.
Decitabine is a DNA methyltransferase inhibitor that causes hypomethylation of DNA, leading to reactivation of silenced tumor suppressor genes and differentiation of myelodysplastic cells.
Decitabine is a DNA methyltransferase inhibitor that causes hypomethylation of DNA, leading to reactivation of silenced tumor suppressor genes and differentiation of myelodysplastic cells. Used for Myelodysplastic syndromes (MDS), Acute myeloid leukemia (AML).
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Oncology Phase 3 boost
+3.0pp
Oncology Phase 3 trials have higher approval rates (~61%) than the cross-industry average due to clearer endpoints and FDA oncology pathway.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | Decitabine/Cedazuridine |
|---|---|
| Also known as | ASTX727, INQOVI |
| Sponsor | Astex Pharmaceuticals, Inc. |
| Drug class | DNA methyltransferase inhibitor; hypomethylating agent |
| Target | DNA methyltransferase (DNMT) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Decitabine is a cytidine analog that incorporates into DNA and inhibits DNA methyltransferase (DNMT), reducing aberrant DNA methylation in cancer cells. This hypomethylating effect reactivates epigenetically silenced genes, including tumor suppressors, and promotes cell differentiation and apoptosis. Cedazuridine is a cytidine deaminase inhibitor that increases decitabine bioavailability by preventing its degradation, allowing for oral administration instead of intravenous infusion.
Approved indications
- Myelodysplastic syndromes (MDS)
- Acute myeloid leukemia (AML)
Common side effects
- Myelosuppression (anemia, thrombocytopenia, neutropenia)
- Nausea and vomiting
- Fatigue
- Diarrhea
- Constipation
- Infection
Key clinical trials
- Venetoclax in Combination With ASTX727 for the Treatment of Chronic Myelomonocytic Leukemia and Other Myelodysplastic Syndrome/Myeloproliferative Neoplasm (PHASE2)
- A Randomized Study of ASTX727 With or Without Iadademstat in Advanced Myeloproliferative Neoplasms (MPNs) (PHASE2)
- Testing the Combination of Targeted Radiotherapy With Anti-Cancer Drugs, Venetoclax and ASTX-727, to Improve Outcomes for Adults With Newly Diagnosed Acute Myeloid Leukemia (PHASE1)
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- 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 (PHASE1)
- Testing the Combination of Belinostat and SGI-110 (Guadecitabine) or ASTX727 for the Treatment of Unresectable and Metastatic Conventional Chondrosarcoma (PHASE2)
- Testing the Combination of Nivolumab and ASTX727 for Relapsed or Refractory B-Cell Lymphoma (PHASE1)
- Comparing New Treatments for People With Newly Diagnosed Acute Myeloid Leukemia That Has an IDH2 Gene Change (A MyeloMATCH Treatment Trial) (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Decitabine/Cedazuridine CI brief — competitive landscape report
- Decitabine/Cedazuridine updates RSS · CI watch RSS
- Astex Pharmaceuticals, Inc. portfolio CI
Frequently asked questions about Decitabine/Cedazuridine
What is Decitabine/Cedazuridine?
How does Decitabine/Cedazuridine work?
What is Decitabine/Cedazuridine used for?
Who makes Decitabine/Cedazuridine?
Is Decitabine/Cedazuridine also known as anything else?
What drug class is Decitabine/Cedazuridine in?
What development phase is Decitabine/Cedazuridine in?
What are the side effects of Decitabine/Cedazuridine?
What does Decitabine/Cedazuridine target?
Related
- Drug class: All DNA methyltransferase inhibitor; hypomethylating agent drugs
- Target: All drugs targeting DNA methyltransferase (DNMT)
- Manufacturer: Astex Pharmaceuticals, Inc. — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Myelodysplastic syndromes (MDS)
- Indication: Drugs for Acute myeloid leukemia (AML)
- Also known as: ASTX727, INQOVI
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing