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Cytarabine HD
Cytarabine HD is a Nucleoside analog; antimetabolite Small molecule drug developed by Gruppo Italiano Malattie EMatologiche dell'Adulto. It is currently in Phase 3 development for Acute myeloid leukemia (AML), Acute lymphoblastic leukemia (ALL), Lymphoma.
Cytarabine is a cytidine analog that inhibits DNA synthesis by being incorporated into DNA and blocking chain elongation, leading to cell death.
Cytarabine HD is used in the treatment of various conditions, including Central Nervous System Lymphoma, Lymphoma, Pediatric Acute Myeloid Leukemia, and Stage I and Stage II Contiguous Mantle Cell Lymphoma. It is administered as part of immunochemotherapy regimens, often in combination with other medications such as Rituximab, HD-MTX, Cyclophosphamide, and Ifosfamide.
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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 | Cytarabine HD |
|---|---|
| Sponsor | Gruppo Italiano Malattie EMatologiche dell'Adulto |
| Drug class | Nucleoside analog; antimetabolite |
| Target | DNA polymerase; incorporated into DNA |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Cytarabine (ara-C) is a nucleoside analog that mimics cytidine and is phosphorylated intracellularly to its active triphosphate form. This active metabolite is incorporated into DNA during synthesis, causing chain termination and inhibition of DNA polymerase. It is primarily used in hematologic malignancies where rapidly dividing cells are particularly susceptible to its cytotoxic effects.
Approved indications
- Acute myeloid leukemia (AML)
- Acute lymphoblastic leukemia (ALL)
- Lymphoma
- Chronic myeloid leukemia (CML) in blast phase
Common side effects
- Myelosuppression (neutropenia, thrombocytopenia, anemia)
- Nausea and vomiting
- Mucositis
- Hepatotoxicity
- Neurotoxicity (at high doses)
- Infection
Key clinical trials
- Chemotherapy With Targeted-Immunotherapy for Newly Diagnosed Ph+ ALL (NA)
- OptiMATe: De-escalated Induction Treatment in Primary CNS Lymphoma (PHASE3)
- ABL/JAK Inhibitors With Chemotherapy and Venetoclax for Ph-like ALL (NA)
- Auto BMT for Non-M3 AML in 1st Remission in Pts </=60y of Age Using Busulfan/FTBI & VP16 as a Prep R (PHASE2)
- Venetoclax Combined With Azacitidine, Chidamide, Vindesine, and Dexamethasone in Newly Diagnosed ETP-ALL Like Patients (PHASE2)
- New Combination of Chemoimmunotherapy for Systemic B-cell Lymphoma With Central Nervous System Involvement (PHASE2)
- Treatment of Pediatric Very High-risk Acute Lymphoblastic Leukemia in Korea (PHASE2)
- Treatment Protocol for Children and Adolescents With Acute Lymphoblastic Leukemia - AIEOP-BFM ALL 2017 (PHASE3)
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:
- Cytarabine HD CI brief — competitive landscape report
- Cytarabine HD updates RSS · CI watch RSS
- Gruppo Italiano Malattie EMatologiche dell'Adulto portfolio CI
Frequently asked questions about Cytarabine HD
What is Cytarabine HD?
How does Cytarabine HD work?
What is Cytarabine HD used for?
Who makes Cytarabine HD?
What drug class is Cytarabine HD in?
What development phase is Cytarabine HD in?
What are the side effects of Cytarabine HD?
What does Cytarabine HD target?
Related
- Drug class: All Nucleoside analog; antimetabolite drugs
- Target: All drugs targeting DNA polymerase; incorporated into DNA
- Manufacturer: Gruppo Italiano Malattie EMatologiche dell'Adulto — 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 Lymphoma
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing