CLINICAL TRIALS PROFILE FOR CYTARABINE; DAUNORUBICIN

505(b)(2) Clinical Trials for cytarabine; daunorubicin

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT04992949 ↗	Evaluation of CPX-351 Monotherapy in Acute Myeloid Leukemia Secondary to Myeloproliferative Neoplasm	Not yet recruiting	Acute Leukemia French Association	Phase 2	2021-10-01	The three classic myeloproliferative neoplasms (MPNs) include polycythemia Vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF). The natural history of these MPNs is the possible progression to acute myeloid leukemia (MPN-blast phase) at variable percentage depending the entity. Leukemic transformation of MPN occurs in 8% to 23% of primary myelofibrosis (PMF) patients in the first 10 years after diagnosis and in 4% to 8% of polycythemia vera (PV) and essential thrombocytosis (ET) patients within 18 years after diagnosis. The risk for leukemic transformation is increased by exposure to cytotoxic chemotherapy. The molecular pathogenesis of MPN-blast phase remains an area of active research. The prognosis of blast phase MPNs is very poor : approximately 50% of the patients are deemed eligible for intensive treatment (ie. conventional induction chemotherapy regimen with anthracyclines and cytarabine). The patients who are not fit for such intensive treatment approach due to age or comorbidities, are treated with Hypomethylating agents, low dose palliative chemotherapy, or supportive care. Nevertheless, there is a need for more effective and better tolerated treatment approaches in order to increase the response rate and hence, the transplant rates which should translate into improved survival. CPX-351 is a new formulation of cytarabine and daunorubicin encapsulated at a fixed 5:1 molar-ratio in liposomes that exploits molar ratio-dependent drug-drug synergy to enhance antileukemic efficacy. Based on similarities between post-myelodysplastic syndrome (MDS) and post-MPN secondary AML in terms of disease resistance to chemotherapy, of fragile patient profile, The hypotheses made is that CPX-351 may improve the results of induction chemotherapy without increasing its toxicity and therefore may increase the proportion of patients who could benefit from an allogeneic Stem Cell Transplantation (SCT).
New Formulation	NCT04992949 ↗	Evaluation of CPX-351 Monotherapy in Acute Myeloid Leukemia Secondary to Myeloproliferative Neoplasm	Not yet recruiting	French Intergroup of Myeloproliferative syndromes	Phase 2	2021-10-01	The three classic myeloproliferative neoplasms (MPNs) include polycythemia Vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF). The natural history of these MPNs is the possible progression to acute myeloid leukemia (MPN-blast phase) at variable percentage depending the entity. Leukemic transformation of MPN occurs in 8% to 23% of primary myelofibrosis (PMF) patients in the first 10 years after diagnosis and in 4% to 8% of polycythemia vera (PV) and essential thrombocytosis (ET) patients within 18 years after diagnosis. The risk for leukemic transformation is increased by exposure to cytotoxic chemotherapy. The molecular pathogenesis of MPN-blast phase remains an area of active research. The prognosis of blast phase MPNs is very poor : approximately 50% of the patients are deemed eligible for intensive treatment (ie. conventional induction chemotherapy regimen with anthracyclines and cytarabine). The patients who are not fit for such intensive treatment approach due to age or comorbidities, are treated with Hypomethylating agents, low dose palliative chemotherapy, or supportive care. Nevertheless, there is a need for more effective and better tolerated treatment approaches in order to increase the response rate and hence, the transplant rates which should translate into improved survival. CPX-351 is a new formulation of cytarabine and daunorubicin encapsulated at a fixed 5:1 molar-ratio in liposomes that exploits molar ratio-dependent drug-drug synergy to enhance antileukemic efficacy. Based on similarities between post-myelodysplastic syndrome (MDS) and post-MPN secondary AML in terms of disease resistance to chemotherapy, of fragile patient profile, The hypotheses made is that CPX-351 may improve the results of induction chemotherapy without increasing its toxicity and therefore may increase the proportion of patients who could benefit from an allogeneic Stem Cell Transplantation (SCT).
New Formulation	NCT04992949 ↗	Evaluation of CPX-351 Monotherapy in Acute Myeloid Leukemia Secondary to Myeloproliferative Neoplasm	Not yet recruiting	French Innovative Leukemia Organisation	Phase 2	2021-10-01	The three classic myeloproliferative neoplasms (MPNs) include polycythemia Vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF). The natural history of these MPNs is the possible progression to acute myeloid leukemia (MPN-blast phase) at variable percentage depending the entity. Leukemic transformation of MPN occurs in 8% to 23% of primary myelofibrosis (PMF) patients in the first 10 years after diagnosis and in 4% to 8% of polycythemia vera (PV) and essential thrombocytosis (ET) patients within 18 years after diagnosis. The risk for leukemic transformation is increased by exposure to cytotoxic chemotherapy. The molecular pathogenesis of MPN-blast phase remains an area of active research. The prognosis of blast phase MPNs is very poor : approximately 50% of the patients are deemed eligible for intensive treatment (ie. conventional induction chemotherapy regimen with anthracyclines and cytarabine). The patients who are not fit for such intensive treatment approach due to age or comorbidities, are treated with Hypomethylating agents, low dose palliative chemotherapy, or supportive care. Nevertheless, there is a need for more effective and better tolerated treatment approaches in order to increase the response rate and hence, the transplant rates which should translate into improved survival. CPX-351 is a new formulation of cytarabine and daunorubicin encapsulated at a fixed 5:1 molar-ratio in liposomes that exploits molar ratio-dependent drug-drug synergy to enhance antileukemic efficacy. Based on similarities between post-myelodysplastic syndrome (MDS) and post-MPN secondary AML in terms of disease resistance to chemotherapy, of fragile patient profile, The hypotheses made is that CPX-351 may improve the results of induction chemotherapy without increasing its toxicity and therefore may increase the proportion of patients who could benefit from an allogeneic Stem Cell Transplantation (SCT).

All Clinical Trials for cytarabine; daunorubicin

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002471 ↗	Combination Chemotherapy in Treating Patients With Acute B-Lymphoblastic Leukemia or Non-Hodgkin's Lymphoma	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	1990-02-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients who have acute B-lymphoblastic leukemia or recurrent non-Hodgkin's lymphoma.
NCT00002499 ↗	Combination Chemotherapy in Treating Children With Relapsed Acute Lymphocytic Leukemia	Unknown status	Grupo Argentino de Tratamiento de la Leucemia Aguda	Phase 2/Phase 3	1990-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II/III trial to study the effectiveness of combination chemotherapy in treating children with relapsed acute lymphocytic leukemia.
NCT00002514 ↗	Stem Cell Transplantation Compared With Standard Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia in First Remission	Completed	Medical Research Council	Phase 3	1993-04-01	RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with allogeneic or autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. It is not yet known whether stem cell transplantation is more effective than standard chemotherapy in treating acute lymphoblastic leukemia. PURPOSE: This randomized phase III trial is studying how well stem cell transplantation works compared to standard combination chemotherapy in treating patients with acute lymphoblastic leukemia in first remission.
NCT00002514 ↗	Stem Cell Transplantation Compared With Standard Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia in First Remission	Completed	National Cancer Institute (NCI)	Phase 3	1993-04-01	RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with allogeneic or autologous stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. It is not yet known whether stem cell transplantation is more effective than standard chemotherapy in treating acute lymphoblastic leukemia. PURPOSE: This randomized phase III trial is studying how well stem cell transplantation works compared to standard combination chemotherapy in treating patients with acute lymphoblastic leukemia in first remission.

Clinical Trial Conditions for cytarabine; daunorubicin

Condition Name

Condition Name for cytarabine; daunorubicin
Intervention	Trials
Acute Myeloid Leukemia	94
Leukemia	81
Untreated Adult Acute Myeloid Leukemia	23
Acute Lymphoblastic Leukemia	19

Condition MeSH

Condition MeSH for cytarabine; daunorubicin
Intervention	Trials
Leukemia	254
Leukemia, Myeloid, Acute	192
Leukemia, Myeloid	164
Precursor Cell Lymphoblastic Leukemia-Lymphoma	84

Clinical Trial Locations for cytarabine; daunorubicin

Trials by Country

Trials by Country for cytarabine; daunorubicin
Location	Trials
Canada	231
Japan	54
China	40
Germany	39
Italy	37

Trials by US State

Trials by US State for cytarabine; daunorubicin
Location	Trials
New York	91
California	90
Illinois	86
Ohio	84
Texas	81

Clinical Trial Progress for cytarabine; daunorubicin

Clinical Trial Phase

Clinical Trial Phase for cytarabine; daunorubicin
Clinical Trial Phase	Trials
PHASE3	4
PHASE2	16
PHASE1	5

Clinical Trial Status

Clinical Trial Status for cytarabine; daunorubicin
Clinical Trial Phase	Trials
Completed	128
Recruiting	69
Active, not recruiting	31

Clinical Trial Sponsors for cytarabine; daunorubicin

Sponsor Name

Sponsor Name for cytarabine; daunorubicin
Sponsor	Trials
National Cancer Institute (NCI)	122
Children's Oncology Group	32
Jazz Pharmaceuticals	19

Sponsor Type

Sponsor Type for cytarabine; daunorubicin
Sponsor	Trials
Other	326
NIH	125
Industry	117

Last updated: May 21, 2026

CYTARABINE + DAUNORUBICIN Clinical Trials Update, Market Analysis, and Exclusivity/Generic Risk Projection

Executive summary: Cytarabine plus daunorubicin remains a high-value backbone in acute myeloid leukemia (AML) induction. Clinical development and commercial outlook are driven more by new combinations, altered dosing schedules, and next-generation induction strategies than by “new” cytarabine or daunorubicin themselves. Market value is concentrated in hospitals and academic centers in the US and EU, with pricing and access shaped by shortages, contract pharmacy dynamics, and generic penetration of long-established cytarabine and daunorubicin products. Near-term exclusivity risk is low at the active-ingredient level for most geographies, but mid-term competitive intensity can rise if sponsors’ label expansions or formulation/device IP around specific products constrain interchangeable generic entry. The key investment and licensing question is whether any current trials target a distinct patient subpopulation or a regimen with differentiating endpoints that could support an expanded indication and payer adoption.

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What clinical trials are evaluating cytarabine plus daunorubicin in AML right now?

Featured answer: Current trial activity around cytarabine plus daunorubicin is dominated by regimen optimization: doublets with targeted agents, changes in induction intensity, and consolidation/maintenance sequencing. Enrollment expansion tends to be faster when sponsors use the cytarabine/daunorubicin backbone as a platform rather than pursuing a standalone formulation of either drug.

Trial types that show up most often with this backbone

• Platform combination trials: cytarabine/daunorubicin plus a targeted small molecule or antibody-conjugate class agent.
• Fitness and age-stratified induction: modifications of daunorubicin exposure or cytarabine dose intensity in older/unfit AML cohorts.
• MRD-guided strategies: induction response or MRD kinetics driving consolidation intensity and transplant decisions.
• Device and administration logistics: trials that test schedule changes that impact adherence or toxicity management, especially around cytarabine schedule and antiemetic/supportive regimens.

What to track for “meaningful” clinical readouts

• Composite remission endpoints that are regimen-sensitive in AML: CR/CRi, time to count recovery, induction mortality.
• Safety signals: febrile neutropenia, mucositis, cardiotoxicity and hepatic AEs attributable to cumulative anthracycline exposure.
• Post-remission impact: relapse-free survival (RFS) and overall survival (OS) after consolidation and transplant.
• Subgroup performance: older/unfit AML, adverse-risk cytogenetics, and TP53-mutant outcomes.

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Which active ingredient risks are most relevant: cytarabine vs daunorubicin patents and formulation IP?

Featured answer: For commercial risk, most patent barriers at the active-ingredient level are expired in major markets. The live IP risk usually sits in specific branded products (formulation, dosing regimen patents tied to a marketed schedule, or manufacturing process claims) rather than the core chemistry of cytarabine or daunorubicin.

How IP typically clusters for legacy AML backbones

• Drug substance and core compositions: largely expired.
• Specific dosage form compositions: viscosity modifiers, excipients, stability and reconstitution-related compositions for particular products.
• Method-of-use: regimen-specific claims, such as timing, dose sequencing, or a defined induction-and-consolidation workflow paired with particular patient characteristics.
• Manufacturing/process: controls tied to particle size/impurity profiles and sterile filling.

Practical consequence for freedom-to-operate

• Generic entry is often feasible at the API level but may require product-specific design-around for excipients, stability performance, or dosing schedule claims.
• Labeling and interchangeability: even when a generic is approved, market penetration depends on local hospital formularies, procurement contracts, and supply continuity.

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What is the Orange Book status of cytarabine and daunorubicin products, and where does it matter for exclusivity?

Featured answer: The Orange Book listing for cytarabine and daunorubicin is typically dominated by multiple approved ANDAs with limited ongoing exclusivity at the active-ingredient level. The practical exclusivity remaining tends to be tied to specific branded NDA or supplemental patents for particular dosage forms, not blanket compound exclusivity.

Where exclusivity tends to matter anyway

• Single-source branded presentations that retain formulary preference due to stability, packaging, or clinician trust.
• Hospital procurement behavior: conversion cycles and procurement rules can delay switching even if patents expire.
• Bundled procurement of induction kits: some institutions standardize on a single induction regimen kit, slowing dispersion even after patent loss.

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When do cytarabine or daunorubicin lose exclusivity, and what timeline matters for generic launches?

Featured answer: In most markets, active-ingredient exclusivity for cytarabine and daunorubicin is already expired; the remaining timeline risk is product-specific patent expiration and any method-of-use or formulation claims still active on branded schedules.

Exclusivity timeline framework (how to project)

• Step 1: Identify the specific marketed product (NDA/label presentation, strength, packaging).
• Step 2: Map Orange Book “patent lives” to the product (drug substance, drug product, method-of-use).
• Step 3: Translate into generic timing using Paragraph IV opportunities and potential litigation stay periods.
• Step 4: Convert into hospital uptake timeline (contracting and substitution lead time).

Generic launch projection logic for legacy oncology drugs

• Approval date is not the adoption date. Adoption often lags by 3 to 18 months due to procurement cycles.
• Tender cycles and supply stability are decisive if the institution has experienced shortages.

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Are there any Paragraph IV challenges or patent litigations involving cytarabine plus daunorubicin products?

Featured answer: Litigation in cytarabine/daunorubicin typically occurs around product-specific patents. At the class level, the volume of litigation is lower than for newer oncology biologics or novel small molecules because many foundational patents are already out of date.

How to assess litigation impact on market access

• Paragraph IV events that attach to specific strengths and presentations can delay a subset of supply while generic entrants get injunction threats.
• Settlement agreements commonly lead to “at-risk” launch timing that is earlier than the final patent expiry if the settlement permits launch.
• Injunction scope: if a court limits to a particular dosage form, hospitals may still switch other strengths.

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What formulations are protected for cytarabine and daunorubicin, and what products have the most IP leverage?

Featured answer: The highest residual formulation leverage tends to be associated with specific branded dosage forms, stability and reconstitution solutions, and packaging designed for sterile handling.

Formulation and administration topics that can carry IP

• Stability during storage and after reconstitution
• Excipient systems affecting solubility and local tolerability
• Packaging formats that reduce dosing errors or improve delivery consistency
• Manufacturing impurity profiles that protect a specific acceptance specification

Market consequence

Even when two products are therapeutically equivalent, procurement may prefer the product with:

• more predictable supply
• fewer preparation-related incidents
• established performance with institutional protocols

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How does the cytarabine plus daunorubicin regimen compare with other AML induction standards (and what does that do to demand)?

Featured answer: Demand for cytarabine/daunorubicin is affected less by “superiority” and more by label positioning and clinician preference among alternatives like venetoclax-based regimens in unfit patients, and targeted/novel agent combinations in fitness strata. In fit patients, standard anthracycline plus cytarabine induction remains an anchor in many settings.

Competitive substitution pressures

• Venetoclax + hypomethylating agents: often shifts volume away from intensive induction in older/unfit AML.
• CPX-351 (liposomal daunorubicin/cytarabine): competes for induction share by targeting improved outcomes in high-risk AML subgroups.
• Other targeted induction combinations: can redirect usage depending on molecular stratification and reimbursement.

Commercial impact channels

• Guideline alignment: if guidelines favor a different regimen for a subpopulation, the backbone sees step-down in that cohort.
• Real-world treatment mix: varies by region due to payer coverage and hospital capabilities for ICU and transplant pathways.

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Market analysis: where are sales concentrated and how is pricing trending for cytarabine and daunorubicin?

Featured answer: Market sales are concentrated in institutional procurement, especially induction and consolidation for AML. Pricing trends are driven by generic competition, tender-based contracts, and periodic supply disruptions.

Demand drivers

• AML incidence and treatment intensity: number of eligible patients undergoing induction.
• Protocol adherence: cytarabine/daunorubicin is embedded in many induction pathways.
• Hospital formularies: switching costs favor incumbents even post-patent expiry.

Pricing dynamics

• Generic substitution compresses net price over time.
• Branded products can retain premium if procurement contracts lock in specific vendors or if supply issues make alternative sourcing risky.
• Supply constraints can temporarily lift pricing and stabilize margins for available suppliers.

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What market projection scenarios are most plausible over the next 5 years?

Featured answer: Base-case growth is modest because the drugs are mature and heavily genericized. Upside scenarios depend on (1) increased AML incidence, (2) regimen-specific label expansions for particular cytarabine/daunorubicin delivery formats, or (3) reduced substitution in certain geographies due to supply constraints.

Scenario framework (directional, decision-oriented)

• Base case (stable to low growth):
  • AML regimen mix shifts slowly toward non-intensive options for unfit patients.
  • Fit-patient intensive induction continues as a stable share.
• Downside case (share erosion):
  • Venetoclax-based regimens gain further coverage and guideline favorability in broader unfit populations.
  • CPX-351 or other novel combinations capture additional high-risk induction share.
• Upside case (capacity-constrained incumbents):
  • Supply disruptions persist for certain presentations, reducing substitution.
  • Institutions delay switching and continue branded/preferred generics through procurement cycles.

What to use for monitoring

• Monthly supply availability by strength and presentation.
• Hospital tender changes for induction kits.
• Share of treated AML patients by regimen class in claims databases.

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Biosimilar risk exists for this regimen?

Featured answer: No. Cytarabine and daunorubicin are small-molecule drugs, not biologics, so “biosimilar risk” is not the right framework. The competitive risk is generic drug substitution and product-specific formulation IP.

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Which companies are positioned to benefit or to be displaced in cytarabine and daunorubicin supply?

Featured answer: Benefit typically accrues to manufacturers with (1) reliable sterile supply, (2) strong tender relationships, and (3) presentations that are preferred for reconstitution and stability. Displacement risk targets suppliers with weaker supply continuity or with products that lose procurement preference after contract renewal.

Practical competitive map

• US and EU generic injectables suppliers: typically compete on availability, pricing, and compliance performance.
• Branded legacy suppliers: maintain a hold if procurement contracts and clinician habit persist despite generic options.

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What is the biggest commercial IP barrier to generic entry: manufacturing constraints or patent claims?

Featured answer: For mature cytarabine and daunorubicin, commercial barriers are often more operational than legal. Manufacturing compliance, sterile filling capacity, and impurity control tend to matter as much as patents, especially when supply is constrained.

Decision lens for investors and licensors

• If patents are expired, the limiting factor is manufacturing scale and regulatory readiness.
• If patents are active for a specific formulation, then litigation and design-around determine entry timing.

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Key Takeaways

• Cytarabine plus daunorubicin remains a central AML induction backbone, but clinical and commercial momentum depends on platform combinations, schedule optimization, and patient selection rather than on new chemistry.
• Patent exclusivity at the active-ingredient level is largely exhausted in major markets; the remaining risk is product-specific formulation or method-of-use IP attached to specific marketed presentations.
• Market growth is modest and mostly constrained by generic penetration, institutional procurement behavior, and regimen-share shifts toward non-intensive options for unfit patients.
• Competitive dynamics are operational as much as legal: supply continuity and procurement relationships often determine net share more than incremental IP.

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FAQs

1. Which AML patient groups are most likely to receive cytarabine plus daunorubicin rather than venetoclax-based regimens?
2. What endpoints in induction AML trials most directly predict adoption of cytarabine/daunorubicin-based combinations?
3. How do hospital tender cycles affect the speed of generic substitution for cytarabine and daunorubicin products?
4. Which formulation characteristics (stability, reconstitution, packaging) most influence institutional product preference?
5. How does CPX-351’s liposomal daunorubicin/cytarabine positioning change demand for conventional cytarabine plus daunorubicin?

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References

1. [No citable sources were provided in the prompt, and no reliable, product-specific clinical trial or Orange Book dataset was supplied to support citation-quality assertions.]