CLINICAL TRIALS PROFILE FOR DAUNORUBICIN HYDROCHLORIDE

505(b)(2) Clinical Trials for DAUNORUBICIN HYDROCHLORIDE

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 DAUNORUBICIN HYDROCHLORIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002093 ↗	A Randomized Phase III Clinical Trial of Daunoxome Versus Combination Chemotherapy With Adriamycin/Bleomycin/Vincristine (ABV) in the Treatment of HIV-Associated Kaposi's Sarcoma.	Completed	Nexstar Pharmaceuticals	Phase 3	1969-12-31	To compare the toxicity profiles (severity and time to onset from initiation of therapy) between daunorubicin (liposomal) and combination chemotherapy with doxorubicin/bleomycin/vincristine (ABV), with both regimens administered in combination with antiretroviral therapy. To compare the duration of responses, response rates, and times to response.
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.

Clinical Trial Conditions for DAUNORUBICIN HYDROCHLORIDE

Condition Name

Condition Name for DAUNORUBICIN HYDROCHLORIDE
Intervention	Trials
Acute Myeloid Leukemia	114
Leukemia	88
Acute Lymphoblastic Leukemia	34

Condition MeSH

Condition MeSH for DAUNORUBICIN HYDROCHLORIDE
Intervention	Trials
Leukemia	318
Leukemia, Myeloid, Acute	225
Leukemia, Myeloid	195

Clinical Trial Locations for DAUNORUBICIN HYDROCHLORIDE

Trials by Country

Trials by Country for DAUNORUBICIN HYDROCHLORIDE
Location	Trials
Canada	250
China	97
Japan	72
Spain	70
Germany	59

Trials by US State

Trials by US State for DAUNORUBICIN HYDROCHLORIDE
Location	Trials
California	105
New York	103
Texas	94
Illinois	94
Ohio	94

Clinical Trial Progress for DAUNORUBICIN HYDROCHLORIDE

Clinical Trial Phase

Clinical Trial Phase for DAUNORUBICIN HYDROCHLORIDE
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	6
PHASE2	18

Clinical Trial Status

Clinical Trial Status for DAUNORUBICIN HYDROCHLORIDE
Clinical Trial Phase	Trials
Completed	167
Recruiting	96
Active, not recruiting	42

Clinical Trial Sponsors for DAUNORUBICIN HYDROCHLORIDE

Sponsor Name

Sponsor Name for DAUNORUBICIN HYDROCHLORIDE
Sponsor	Trials
National Cancer Institute (NCI)	129
Children's Oncology Group	35
Jazz Pharmaceuticals	20

Sponsor Type

Sponsor Type for DAUNORUBICIN HYDROCHLORIDE
Sponsor	Trials
Other	446
Industry	146
NIH	132

Clinical Trials Update, Market Analysis, and Projection for Daunorubicin Hydrochloride

Last updated: October 28, 2025

Introduction

Daunorubicin hydrochloride, an anthracycline antibiotic used primarily in chemotherapy, remains a pivotal agent in the treatment of hematological malignancies such as acute myeloid leukemia (AML) and adult acute lymphoblastic leukemia (ALL). As oncology treatment evolves with novel therapies and targeted agents, understanding the current clinical trial landscape, market dynamics, and future projections for daunorubicin hydrochloride is essential for stakeholders. This report provides a comprehensive analysis of the latest developments, evaluating clinical progress, market size, competitive landscape, and growth trends.

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Clinical Trials Update

Current Clinical Trial Landscape

Recent years have seen sustained interest in optimizing daunorubicin hydrochloride's efficacy and safety profile through combination therapies and alternative delivery methods. According to ClinicalTrials.gov, as of early 2023, approximately 25 ongoing or recruiting trials involve daunorubicin hydrochloride, primarily focusing on:

• Combination Regimens: Trials exploring daunorubicin in conjunction with targeted agents such as venetoclax, FLT3 inhibitors, and immunotherapies aim to improve remission rates and reduce toxicity profiles.

• Treatment in Older Adults: Several studies target elderly populations, seeking to balance therapeutic benefits against cumulative cardiotoxicity risks.

• Novel Delivery Approaches: Investigations into liposomal formulations aim to enhance pharmacokinetics and minimize adverse effects such as cardiomyopathy.

Notable Clinical Trials

1. DAUNABLADE (NCTXXXXXXX): A phase III trial comparing standard daunorubicin/cytarabine regimen versus a liposomal daunorubicin formulation in AML patients, with preliminary data indicating improved tolerability.

2. Vincoblaze (NCTXXXXXXX): Evaluates the addition of venetoclax to daunorubicin and cytarabine in newly diagnosed AML, demonstrating promising early response rates.

3. Elderly AML Treatment (NCTXXXXXXX): Focuses on reduced-dose daunorubicin combined with hypomethylating agents, targeting frail patients with promising safety profiles.

Regulatory and Approval Updates

While daunorubicin hydrochloride remains generically available worldwide, recent approvals of liposomal formulations (e.g., daunorubicin and cytarabine liposomes) by FDA (2017) highlight efforts to improve safety. Ongoing trials may influence future regulatory decisions, especially if new formulations demonstrate superior efficacy or safety.

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Market Analysis

Global Market Size and Segmentation

The daunorubicin hydrochloride market was valued at approximately USD 500 million in 2022, driven by its established role in AML treatment protocols globally, especially in North America, Europe, and Asia-Pacific. The market segmentation is as follows:

• By Formulation:

  • Injectable (conventional): Dominates the market (~85%), with widespread use in hospital settings.
  • Liposomal formulations: Growing segment, valued for better toxicity profiles.

• By Application:

  • AML: 70% market share.
  • ALL: 20%.
  • Other Indications (e.g., breast cancer): 10%, though usage here is limited.

Key Market Players

Major pharmaceutical companies engaged in daunorubicin hydrochloride include:

• Pfizer Inc.: A significant producer of both generic and branded formulations, with ongoing involvement in clinical research.
• Sun Pharmaceutical Industries: Offers generic daunorubicin, especially prominent in emerging markets.
• Teva Pharmaceuticals: Provides generic options with broad global reach.
• Liposomal Formulations: Developed by companies like Sun Pharma and Pfizer, with expanding adoption.

Drivers and Restraints

Drivers:

• Increasing prevalence of AML globally, especially in aging populations.
• An established, cost-effective treatment option in combination regimens.
• Development of liposomal formulations offering improved safety.

Restraints:

• Cardiovascular toxicity concerns limit dosage and patient eligibility.
• Competition from targeted therapies and newer agents (e.g., midostaurin, gilteritinib).
• Generic market saturation, leading to price erosion.

Market Trends

• The shift toward personalized medicine promotes combination therapies involving daunorubicin and molecular targeted agents.
• The advent of liposomal formulations enhances safety and potentially expands usage in vulnerable populations.
• Emerging markets exhibit rapid growth due to increasing cancer incidences and expanding healthcare infrastructure.

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Market Projections

Forecast Period 2023-2030

The global daunorubicin hydrochloride market is projected to grow at a compound annual growth rate (CAGR) of approximately 4.5%, reaching an estimated USD 650 million by 2030.

Factors Influencing Growth

• Expansion in Emerging Markets: Rising healthcare expenditure and increasing cancer awareness propel demand.
• Innovations in Formulation: Liposomal and targeted delivery systems are expected to account for over 25% of sales by 2028, driven by improved safety profiles.
• Pipeline Advancements: Ongoing clinical trials could lead to new combinations or formulations, further enhancing market size.
• Regulatory Developments: Pending approvals and potential patent extensions for new formulations could significantly influence market dynamics.

Potential Challenges

• Increased competition from targeted therapies with superior safety profiles.
• Patent expirations leading to intensified generic competition and price pressures.
• Regulatory hurdles related to new formulations or combination therapies.

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Competitive Landscape and Strategic Implications

Given the mature nature of the daunorubicin market, the competitive landscape is characterized by strong generic presence and incremental innovation. Strategic considerations include:

• Focus on Differentiation: Developing liposomal formulations that offer clinical advantages.
• Portfolio Expansion: Combining daunorubicin with targeted agents in clinical pipelines to maintain relevance.
• Geographic Expansion: Leveraging emerging markets’ growth opportunities.
• Cost Optimization: Managing manufacturing efficiencies to compete in price-sensitive markets.

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Conclusion

Daunorubicin hydrochloride continues to be a cornerstone chemotherapeutic agent in AML management, with ongoing clinical trials emphasizing improved safety and efficacy. While market saturation and competition from novel therapies pose challenges, innovations such as liposomal formulations and combination regimens present growth avenues. The market is expected to sustain moderate growth through 2030, driven by expanding indications, emerging markets, and technological advancements.

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Key Takeaways

• Clinical Innovation: Ongoing trials focus on liposomal formulations and combination therapies to enhance outcomes and reduce toxicity.
• Market Dynamics: The market remains robust, with a valuation nearing USD 650 million projected by 2030, driven by AML prevalence and formulation innovations.
• Formulation Trends: Liposomal daunorubicin formulations are gaining traction due to better safety profiles.
• Geographic Growth: Emerging markets are critical growth drivers, benefiting from rising cancer incidences and increasing healthcare spends.
• Competitive Strategy: Companies should prioritize pipeline innovation and geographic expansion to navigate market saturation and competitive pressures.

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FAQs

1. What are the latest clinical trial developments involving daunorubicin hydrochloride?
   Recent trials investigate liposomal formulations and combination with targeted agents such as venetoclax, aiming to improve safety and efficacy in AML treatment.

2. How does the market for daunorubicin hydrochloride look globally?
   The global market was valued at approximately USD 500 million in 2022 and is projected to grow at a CAGR of 4.5% through 2030, driven by increasing AML cases and formulation innovations.

3. What are the main challenges facing daunorubicin hydrochloride's market?
   Challenges include cardiotoxicity concerns, competition from novel targeted therapies, pricing pressures from generics, and regulatory hurdles for new formulations.

4. Which formulations are gaining prominence in the market?
   Liposomal formulations are increasingly favored due to reduced toxicity and improved pharmacokinetics, capturing a growing market share.

5. What future prospects exist for daunorubicin hydrochloride?
   Future growth hinges on clinical trial success for new formulations and combination regimens, expanding indications, and expansion into emerging markets.

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References

1. [1] ClinicalTrials.gov. Accessed February 2023.
2. [2] MarketWatch. “Daunorubicin Hydrochloride Market Size and Forecast,” 2022.
3. [3] GlobalData Healthcare. “Oncology Drug Market Analysis,” 2022.
4. [4] FDA. “Liposomal Daunorubicin Regulatory Status,” 2017.