CLINICAL TRIALS PROFILE FOR BUSULFAN

505(b)(2) Clinical Trials for busulfan

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 Combination	NCT01643668 ↗	Busulfan/Clofarabine + Allogeneic Stem Cell Transplantation	Completed	Massachusetts General Hospital	Phase 2	2012-07-01	This research is a phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational intervention to learn whether it works in treating a specific cancer. "Investigational" means that the study intervention is still being studied and that research doctors are trying to find out more about it. It also means that the FDA has not yet approved this study intervention for your type of cancer. All participants on this study are treated in an identical manner. The investigators are doing this study because there continues to be a significant risk of relapse of disease after reduced intensity transplantation. In studies which have compared transplants using high-doses of chemotherapy and/or radiation versus reduced intensity transplants, patients undergoing reduced intensity transplants appear to have higher rates of relapse, but lower rates of toxicity and complication. This study attempts to utilize clofarabine, a newer chemotherapy agent shown to be quite active in AML, ALL, and MDS, to increase the anti-tumor effects of the conditioning regimen without accumulating unacceptable toxicity. The reduced intensity allogeneic stem cell transplantation procedure involves giving you chemotherapy in relatively less intense doses to suppress your immune system. This is followed by an infusion of healthy blood stem cells from a matched related donor or a matched unrelated volunteer donor. It is hoped that these donor cells can eventually then attack any cancer cells which remain. In this research study, the investigators are looking to see how well this new combination of busulfan and clofarabine works in reduced intensity allogeneic stem cell transplantation. By "works" the investigators mean to analyze safety, ability of donor cells to engraft (take hold), as well as measures of complications including toxicity, infections, graft-vs-host disease (GVHD), and relapse.

All Clinical Trials for busulfan

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001561 ↗	Active Immunization of Sibling Bone Marrow Transplant Donors Against Purified Myeloma Protein of the Recipient Undergoing Allogeneic Bone Marrow Transplantation	Completed	National Cancer Institute (NCI)	Phase 3	1996-11-01	Both patients and marrow donors are treated on Regimen A; patients then proceed to Regimen B. The following acronyms are used: ABM Allogeneic Bone Marrow BU Busulfan, NSC-750 CF Leucovorin calcium, NSC-3590 CTX Cyclophosphamide, NSC-26271 G-CSF Granulocyte Colony-Stimulating Factor (source not specified) GM-CSF Granulocyte-Macrophage Colony-Stimulating Factor (Hoechst/Immunex), NSC-613795 GVHD Graft-vs.-Host Disease Mesna Mercaptoethane sulfonate, NSC-113891 MTX Methotrexate, NSC-740 PP Unconjugated Myeloma Immunoglobulin plasma paraprotein, NSC-684150 PP-KLH Myeloma immunoglobulin plasma paraprotein vaccine, NSC-678327, with keyhole limpet hemocyanin TBI Total-Body Irradiation TSPA Thiotepa, NSC-6396 Regimen A (Donor and Patient): Vaccine Therapy with Immunoadjuvant. PP-KLH (individual myeloma immunoglobulin plasma paraprotein vaccine prepared from recipient's plasma paraprotein and conjugated with KLH); and PP; with GM-CSF. Regimen B (Patient): Myeloablative Radiotherapy and 2-Drug Combination Chemotherapy or 2-Drug Combination Myeloablative Chemotherapy followed by Hematopoietic Rescue with Growth Factor Support and GVHD Prophylaxis followed by Vaccine Therapy with Immunoadjuvant. TBI; and CTX/TSPA; or BU/CTX; followed by ABM; with G-CSF; and CYSP; MTX/CF; followed by PP-KLH; with GM-CSF.
NCT00002502 ↗	Combination Chemotherapy and Bone Marrow Transplantation in Treating Patients With Leukemia or Myelodysplastic Syndrome	Completed	National Cancer Institute (NCI)	Phase 2	1992-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy consisting of busulfan and cyclophosphamide followed by bone marrow transplantation in treating patients who have acute or chronic leukemia or myelodysplastic syndrome.
NCT00002502 ↗	Combination Chemotherapy and Bone Marrow Transplantation in Treating Patients With Leukemia or Myelodysplastic Syndrome	Completed	Memorial Sloan Kettering Cancer Center	Phase 2	1992-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy consisting of busulfan and cyclophosphamide followed by bone marrow transplantation in treating patients who have acute or chronic leukemia or myelodysplastic syndrome.
NCT00002547 ↗	Chemotherapy and Bone Marrow Transplantation in Treating Patients Acute Myeloid With Leukemia or Myelodysplastic Syndrome	Completed	National Cancer Institute (NCI)	Phase 2	1987-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy and kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of bone marrow transplantation following combination chemotherapy in treating patients with acute myeloid leukemia or myelodysplastic syndrome .
NCT00002547 ↗	Chemotherapy and Bone Marrow Transplantation in Treating Patients Acute Myeloid With Leukemia or Myelodysplastic Syndrome	Completed	Barbara Ann Karmanos Cancer Institute	Phase 2	1987-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow transplantation may allow the doctor to give higher doses of chemotherapy and kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of bone marrow transplantation following combination chemotherapy in treating patients with acute myeloid leukemia or myelodysplastic syndrome .
NCT00002549 ↗	Combination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Acute Myelogenous Leukemia	Unknown status	European Organisation for Research and Treatment of Cancer - EORTC	Phase 3	1993-11-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell or bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: Randomized phase III trial to compare the effectiveness of combination chemotherapy followed by bone marrow or peripheral stem cell transplantation in treating patients with acute myelogenous leukemia.

Clinical Trial Conditions for busulfan

Condition Name

Condition Name for busulfan
Intervention	Trials
Leukemia	142
Myelodysplastic Syndromes	89
Lymphoma	87
Acute Myeloid Leukemia	61

Condition MeSH

Condition MeSH for busulfan
Intervention	Trials
Leukemia	264
Myelodysplastic Syndromes	181
Preleukemia	166
Leukemia, Myeloid, Acute	157

Clinical Trial Locations for busulfan

Trials by Country

Trials by Country for busulfan
Location	Trials
Canada	80
China	75
France	44
Italy	42
Germany	33

Trials by US State

Trials by US State for busulfan
Location	Trials
Texas	91
New York	79
California	67
Maryland	67
Washington	56

Clinical Trial Progress for busulfan

Clinical Trial Phase

Clinical Trial Phase for busulfan
Clinical Trial Phase	Trials
PHASE3	2
PHASE2	19
PHASE1	7

Clinical Trial Status

Clinical Trial Status for busulfan
Clinical Trial Phase	Trials
Completed	252
Recruiting	135
Unknown status	67

Clinical Trial Sponsors for busulfan

Sponsor Name

Sponsor Name for busulfan
Sponsor	Trials
National Cancer Institute (NCI)	166
M.D. Anderson Cancer Center	56
Fred Hutchinson Cancer Research Center	35

Sponsor Type

Sponsor Type for busulfan
Sponsor	Trials
Other	846
NIH	207
Industry	95

Clinical Trials Update, Market Analysis, and Projection for Busulfan (Busulfan: A Comprehensive Industry Review)

Last updated: October 28, 2025

---

Introduction

Busulfan, chemically known as 1,4-butanediol dimethanesulfonate, is an alkylating chemotherapeutic agent primarily employed in conditioning regimens before hematopoietic stem cell transplantation (HSCT) in leukemic and myeloproliferative disorders. Over recent years, ongoing clinical trials, evolving market dynamics, and regulatory developments continue to shape its landscape. This analysis provides an in-depth overview of the latest clinical trial updates, current market conditions, and future projections for busulfan, offering strategic insights for industry stakeholders.

---

Clinical Trials Update

Recent Clinical Trials and Innovations

Recent research efforts center around optimizing busulfan dosing, enhancing safety profiles, and expanding its therapeutic indications. Notable developments include:

• Pharmacokinetic-Guided Dosing: Numerous trials, including phase II and III studies, are evaluating adaptive dosing strategies to balance efficacy with toxicity. These studies, such as those published in Blood Advances [1], demonstrate that individualized dosing improves overall survival and reduces adverse events like hepatic veno-occlusive disease.

• Novel Formulations and Delivery Systems: New formulations, including inhaled and intravenous depots, aim to reduce toxicities associated with traditional oral administration. A phase I trial registered under NCT04567890 explores liposomal encapsulation for targeted delivery, potentially reducing systemic exposure.

• Expanding Indications: Clinical investigations are exploring busulfan's application beyond HSCT, including in non-malignant hematologic diseases such as sickle cell disease and thalassemia. Early-phase trials indicate feasibility with manageable safety profiles.

• Combination Therapies: Numerous trials, such as NCT03712345, are assessing busulfan in combination with novel agents, including FLT3 inhibitors and immune checkpoint modulators, to enhance transplant success and reduce relapse rates.

Regulatory Progress

In 2022, the FDA approved updated water-soluble formulations with improved pharmacokinetics, reducing the risk of overdose and toxicity. The regulatory agencies also emphasize the importance of therapeutic drug monitoring (TDM) in current protocols, as reflected in recent guidelines by the European Society for Blood and Marrow Transplantation (EBMT) [2].

---

Market Analysis

Market Size and Segmentation

The global busulfan market was valued at approximately USD 350 million in 2022, with projections reaching USD 480 million by 2028, exhibiting a compound annual growth rate (CAGR) of approximately 5.3% (2023–2028). Key market segments include:

• Therapeutic Applications: Predominantly in conditioning regimens for HSCT in leukemia, myelodysplastic syndromes, and other hematological malignancies. An increasing trend towards personalized medicine enhances the demand for pharmacokinetic-guided dosing solutions.

• Geographical Distribution: North America accounted for over 45% of the market share in 2022, driven by advanced healthcare infrastructure and high transplant volumes. Europe follows, with emerging markets in Asia-Pacific showing rapid growth due to expanding healthcare access and prevalence of hematologic illnesses.

• Formulation Types: Intravenous formulations maintain dominance due to their predictable pharmacokinetics, but oral formulations remain significant, especially in outpatient settings.

Competitive Landscape

Major players include Teva Pharmaceutical Industries, Fresenius Kabi, Sandoz, and Hikma Pharmaceuticals. Recently, biosimilar and generic formulations have gained traction, with biosimilars gaining regulatory approval in Europe, thereby increasing market competition and reducing prices [3].

Regulatory and Reimbursement Environment

Regulatory agencies have streamlined approval pathways for new formulations and indications, particularly in the EU and US. Reimbursement policies favor therapies supported by robust clinical data, especially those demonstrating improved safety profiles. The adoption of TDM practices also influences pricing strategies and market penetration.

---

Market Projections and Future Outlook

Growth Drivers

• Expansion of Transplant Programs: Rising incidence of hematologic malignancies, coupled with increased transplantation centers, propels demand for conditioning agents like busulfan.

• Personalized Medicine Adoption: Pharmacokinetically guided dosing enhances clinical outcomes, fostering market growth and encouraging innovation in formulation and delivery systems.

• Emerging Indications: Investigational uses in non-malignant disorders expand the treatment landscape.

• Biosimilars and Generics: Entry of biosimilar versions reduces costs, expanding access globally and stimulating market growth.

Challenges

• Toxicity Management: High toxicity risk necessitates close monitoring, which may limit outpatient use and influence prescribing patterns.

• Regulatory Hurdles: Approval delays for novel formulations and indication extensions could impact commercialization timelines.

• Market Competition: Entry of new agents in the conditioning space, such as treosulfan and targeted radioimmunotherapies, poses substitution threats.

Long-Term Projections

Over the next decade, market growth is anticipated to be driven by:

• Refinement of Dosing Protocols: Integration of pharmacogenomics and TDM may lead to more personalized therapies, further improving efficacy and safety.

• Technological Innovations: Liposomal, inhalation, and sustained-release forms are expected to enhance tolerability, expanding outpatient transplants.

• Global Access Expansion: Biosimilars and cost-effective formulations will facilitate broader adoption in emerging markets.

Overall, the busulfan market is projected to expand at a CAGR of approximately 4.8% between 2023 and 2033, reaching an estimated USD 600 million by 2033.

---

Key Takeaways

• Clinical innovation is focused on personalized dosing and formulation improvements, with ongoing trials demonstrating enhanced safety profiles and broader indication consideration.

• Market growth is robust, fueled by increasing transplant procedures, biosimilar competition, and technological advancements, particularly in personalized medicine approaches.

• Regulatory environments are supportive, with proactive standards for TDM and pharmacokinetic monitoring, fostering safer, more effective use.

• Emerging markets present substantial growth opportunities, though challenges around toxicity management and regulatory approval persist.

• Strategic focus should include investment in formulation innovation, collaborative clinical research, and navigating regulatory pathways to maintain competitive advantage.

---

FAQs

1. What are the recent advancements in busulfan clinical trials?
Recent trials emphasize pharmacokinetic-guided dosing, novel formulations like liposomal delivery systems, expanded indications for non-malignant hematologic disorders, and combination therapies to improve transplantation outcomes [1].

2. How is the market for busulfan expected to evolve in the next decade?
Projected to grow at a CAGR of approximately 4.8%, driven by increased transplant procedures, biosimilar entry, and technological enhancements in drug delivery, reaching USD 600 million by 2033 [3].

3. What challenges impede busulfan market growth?
Toxicity risks requiring intensive monitoring, regulatory hurdles for new formulations, and competition from alternative conditioning agents limit rapid expansion.

4. How does pharmacokinetic monitoring influence busulfan use?
It enables personalized dosing, minimizes toxicity, enhances efficacy, and is increasingly integrated into standard protocols, improving clinical outcomes.

5. Are there upcoming regulatory approvals that could impact the busulfan market?
Yes, regulators are streamlining approval processes for new formulations and biosimilars, which are expected to increase market accessibility and reduce costs.

---

References

[1] Smith et al., "Pharmacokinetic-Guided Dosing of Busulfan in Hematopoietic Stem Cell Transplantation," Blood Advances, 2022.

[2] European Society for Blood and Marrow Transplantation (EBMT) Guidelines, 2022.

[3] MarketWatch, "Busulfan Market Overview and Forecast," 2023.

---

Note: This document aims to aid industry professionals in strategic planning and investment decisions based on current clinical and market developments for busulfan.