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high dose cytarabine
high dose cytarabine is a Nucleoside analog antimetabolite Small molecule drug developed by International Extranodal Lymphoma Study Group (IELSG). It is currently in Phase 3 development for Acute myeloid leukemia (AML), Acute lymphoblastic leukemia (ALL), Primary CNS lymphoma. Also known as: HDAC.
High-dose cytarabine is a nucleoside analog that inhibits DNA synthesis by being incorporated into DNA, leading to cell death in rapidly dividing cells.
High dose cytarabine is used to treat various conditions, including T-cell Lymphoma, Acute Myeloid Leukemia, and Liver Lymphoma, among others. It is often used in combination with other treatments, such as consolidation with ASCT, pembrolizumab, or Acalabrutinib, as part of clinical trials.
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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 | high dose cytarabine |
|---|---|
| Also known as | HDAC |
| Sponsor | International Extranodal Lymphoma Study Group (IELSG) |
| Drug class | Nucleoside analog antimetabolite |
| Target | DNA synthesis (cytidine analog) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Cytarabine is a cytidine analog that is phosphorylated intracellularly and incorporated into DNA during the S phase of the cell cycle, causing chain termination and apoptosis. At high doses, it achieves greater intracellular accumulation and DNA incorporation compared to standard-dose regimens, resulting in enhanced cytotoxic effects against hematologic malignancies. This mechanism makes it particularly effective against leukemias and lymphomas with high proliferation rates.
Approved indications
- Acute myeloid leukemia (AML)
- Acute lymphoblastic leukemia (ALL)
- Primary CNS lymphoma
- Lymphoma (as part of combination chemotherapy regimens)
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia, anemia)
- Neurotoxicity (cerebellar syndrome, encephalopathy)
- Mucositis
- Nausea and vomiting
- Infection
- Hepatotoxicity
- Conjunctivitis
Key clinical trials
- Pediatric-Inspired Regimen Combined With Venetoclax and Immunotherapy for Adult Ph-Negative Acute Lymphoblastic Leukemia (NA)
- Standard-dose vs Intermediate-dose Cytarabine Induction in the Treatment of Acute Myeloid Leukemia With RUNX1-RUNX1T1 (PHASE3)
- 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)
- Phase III Study of Induction and Consolidation Chemotherapy With Venetoclax in Patients With Newly Diagnosed AML or MDS-EB-2 (PHASE3)
- Testing the Effects of Novel Therapeutics for Newly Diagnosed, Untreated Patients With High-Risk Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial) (PHASE2)
- Chemotherapy With Targeted-Immunotherapy for Newly Diagnosed Ph+ ALL (NA)
- Venetoclax, Azacitidine and Liposomal Mitoxantrone for Newly Diagnosed AML (NA)
- A Comparison of Three Chemotherapy Regimens for the Treatment of Patients With Newly Diagnosed Mantle Cell Lymphoma (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:
- high dose cytarabine CI brief — competitive landscape report
- high dose cytarabine updates RSS · CI watch RSS
- International Extranodal Lymphoma Study Group (IELSG) portfolio CI
Frequently asked questions about high dose cytarabine
What is high dose cytarabine?
How does high dose cytarabine work?
What is high dose cytarabine used for?
Who makes high dose cytarabine?
Is high dose cytarabine also known as anything else?
What drug class is high dose cytarabine in?
What development phase is high dose cytarabine in?
What are the side effects of high dose cytarabine?
What does high dose cytarabine target?
Related
- Drug class: All Nucleoside analog antimetabolite drugs
- Target: All drugs targeting DNA synthesis (cytidine analog)
- Manufacturer: International Extranodal Lymphoma Study Group (IELSG) — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Acute myeloid leukemia (AML)
- Indication: Drugs for Acute lymphoblastic leukemia (ALL)
- Indication: Drugs for Primary CNS lymphoma
- Also known as: HDAC
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing