Busulfan - Generic Drug Details

What are the generic drug sources for busulfan and what is the scope of patent protection?

Busulfan is the generic ingredient in three branded drugs marketed by Accord Hlthcare Inc, Actavis, Am Regent, Amneal, Apotex, Arthur Grp, Eugia Pharma, Hospira, Meitheal, Mylan Institutional, Nexus, Pharmascience Inc, Pharmobedient, Prinston Inc, Shilpa, Otsuka Pharm, and Waylis Therap, and is included in seventeen NDAs. Additional information is available in the individual branded drug profile pages.

There are ten drug master file entries for busulfan. Eleven suppliers are listed for this compound.

Summary for busulfan

US Patents:	0
Tradenames:	3
Applicants:	17
NDAs:	17
Drug Master File Entries:	10
Finished Product Suppliers / Packagers:	11
Raw Ingredient (Bulk) Api Vendors:	88
Clinical Trials:	630
Patent Applications:	8,132
Drug Prices:	Drug price trends for busulfan
What excipients (inactive ingredients) are in busulfan?	busulfan excipients list
DailyMed Link:	busulfan at DailyMed

See drug prices for busulfan

Recent Clinical Trials for busulfan

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
City of Hope Medical Center	PHASE1
National Cancer Institute (NCI)	PHASE1
Fred Hutchinson Cancer Center	PHASE2

See all busulfan clinical trials

Pharmacology for busulfan

Drug Class	Alkylating Drug
Mechanism of Action	Alkylating Activity

Medical Subject Heading (MeSH) Categories for busulfan

Alkylating Agents

Antineoplastic Agents, Alkylating

Immunosuppressive Agents

Myeloablative Agonists

Anatomical Therapeutic Chemical (ATC) Classes for busulfan

L01AB	Alkyl sulfonates
L01A	ALKYLATING AGENTS
L01	ANTINEOPLASTIC AGENTS
L	Antineoplastic and immunomodulating agents

Paragraph IV (Patent) Challenges for BUSULFAN

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
BUSULFEX	Injection	busulfan	6 mg/mL	020954	1	2012-12-26

US Patents and Regulatory Information for busulfan

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Eugia Pharma	BUSULFAN	busulfan	INJECTABLE;INJECTION	215102-001	Jun 25, 2024		DISCN	No	No
Apotex	BUSULFAN	busulfan	INJECTABLE;INJECTION	210448-001	May 7, 2019		DISCN	No	No
Pharmascience Inc	BUSULFAN	busulfan	INJECTABLE;INJECTION	207050-001	Mar 24, 2017	AP	RX	No	No

Expired US Patents for busulfan

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Otsuka Pharm	BUSULFEX	busulfan	INJECTABLE;INJECTION	020954-001	Feb 4, 1999	5,430,057*PED
Otsuka Pharm	BUSULFEX	busulfan	INJECTABLE;INJECTION	020954-001	Feb 4, 1999	5,559,148*PED

EU/EMA Drug Approvals for busulfan

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Pierre Fabre Medicament	Busilvex	busulfan	EMEA/H/C/000472Busilvex followed by cyclophosphamide (BuCy2) is indicated as conditioning treatment prior to conventional haematopoietic progenitor cell transplantation (HPCT) in adult patients when the combination is considered the best available option.Busilvex following fludarabine (FB) is indicated as conditioning treatment prior to haematopoietic progenitor cell transplantation (HPCT) in adult patients who are candidates for a reduced-intensity conditioning (RIC) regimen.Busilvex followed by cyclophosphamide (BuCy4) or melphalan (BuMel) is indicated as conditioning treatment prior to conventional haematopoietic progenitor cell transplantation in paediatric patients.	Withdrawn	no	no	no	2003-07-09
Fresenius Kabi Deutschland GmbH	Busulfan Fresenius Kabi	busulfan	EMEA/H/C/002806Busulfan Fresenius Kabi followed by cyclophosphamide (BuCy2) is indicated as conditioning treatment prior to conventional haematopoietic progenitor cell transplantation (HPCT) in adult patients when the combination is considered the best available option.Busulfan Fresenius Kabi followed by cyclophosphamide (BuCy4) or melphalan (BuMel) is indicated as conditioning treatment prior to conventional haematopoietic progenitor cell transplantation in paediatric patients.	Authorised	yes	no	no	2014-09-22

Market Dynamics and Financial Trajectory for the Pharmaceutical Drug: Busulfan

Last updated: July 28, 2025

Introduction

Busulfan, a potent alkylating agent primarily used in hematopoietic stem cell transplantation (HSCT) for chronic myelogenous leukemia (CML) and other hematologic malignancies, has experienced a fluctuating market landscape driven by evolving treatment protocols, patent expirations, and emerging competitors. As a cornerstone in conditioning regimens, its market dynamics are intricately linked to advances in oncology, transplant medicine, and pharmaceutical innovation. This report analyzes current market forces, financial trajectories, and future outlooks for busulfan, providing stakeholders with strategic insights.

Historical Market Overview

Since its FDA approval in 1999, busulfan has maintained a critical role in myeloablative conditioning regimens. The initial market was dominated by generic formulations, with a few branded derivatives like Myleran (glutaraldehyde liquid) and Busulfex (oral and IV formulations from Sanofi). The generic segment captured substantial market share owing to cost advantages, with branded products holding premium positioning due to perceived quality and safety profiles.

The global market for busulfan was estimated at approximately $150 million in 2021, with North America accounting for approximately 60%, driven by high transplantation volumes and robust healthcare infrastructure. Emerging economies in Asia-Pacific and Europe contribute the remaining share, with expected growth owing to expanding transplant programs.

Current Market Drivers

1. Growing Hematologic Oncology Treatments

The increasing incidence of hematological malignancies, such as leukemia and multiple myeloma, underscores the continued demand for conditioning agents. The global leukemia prevalence, projected to reach over 1.5 million cases by 2025, fuels transplantation needs where busulfan remains integral.

2. Advancements in Transplant Protocols and Supportive Care

Enhanced supportive care protocols—improved infection management, toxicity mitigation, and pharmacokinetic monitoring—have increased transplant success rates. Busulfan’s dosing optimization through therapeutic drug monitoring (TDM) reduces adverse effects, making it more attractive.

3. Drug Patent Expirations and Generic Competition

Major patents for branded formulations expired in recent years, leading to increased generic manufacturing. This has driven down prices, expanding access but squeezing profit margins for branded producers. The generic segment now accounts for over 70% of the market.

4. Regulatory Approvals and New Formulations

Recent approvals of intravenous (IV) busulfan formulations, such as Busulfex IV, with improved bioavailability and predictable pharmacokinetics, have expanded market reach. Regulatory advances in biosimilars and generic avenues are likely to intensify competition.

5. Emerging Alternatives in Conditioning Regimens

Novel agents, including targeted therapies and alternative alkylators, are being studied to replace or augment busulfan. While some promising options are still in clinical trials, they pose long-term competitive threats.

Market Challenges and Risks

1. Toxicity Profile and Side Effects

Busulfan’s toxicity—particularly hepatic veno-occlusive disease (VOD), pulmonary fibrosis, and CNS effects—limits its use in certain patient populations. Enhanced monitoring practices mitigate risks but add cost and complexity.

2. Competition from Novel Conditioning Agents

Emerging agents such as treosulfan and targeted immunotherapies could potentially supplant busulfan in some protocols, especially as their safety profiles improve.

3. Pricing Pressures and Healthcare Cost Containment

Payors and healthcare systems emphasize cost-effectiveness, favoring cheaper generics over branded products. Increased use of generics also pressures profit margins for innovators.

4. Supply Chain Disruptions

Manufacturing complexities, especially for IV formulations, coupled with raw material shortages—exacerbated by geopolitical factors—pose supply risks.

Financial Trajectory and Future Outlook

1. Market Growth Projections

The global busulfan market is projected to grow at a compound annual growth rate (CAGR) of approximately 4-6% through 2028. Growth is sustained by rising transplant procedures and technological improvements in drug administration.

2. Impact of Biosimilars and Generics

The proliferation of biosimilar and generic versions will continue to drive down prices, reducing revenue potential for brand manufacturers but expanding overall market volume. According to industry analysts, price reductions could reach 15-25% over the next five years.

3. Strategic Opportunities

• Formulation Innovation: Development of less toxic, more targeted formulations or delivery systems could command premium pricing.
• Enhanced Pharmacokinetic Monitoring: Offering integrated solutions for TDM could expand clinical utility and justify higher pricing.
• Geographic Expansion: Market penetration in emerging economies via price-sensitive formulations will support volume growth.

4. Revenue Forecasts

While current annual revenues are around $150 million, conservative estimates foresee a CAGR of 4-5% in the near term, reaching $200-250 million globally by 2028, contingent on patent strategies, formulation development, and market expansion.

Regulatory Environment and Patent Landscape

Patent expirations for busulfan formulations have catalyzed generic competition, with many markets in the United States, Europe, and Asia adopting biosimilar entry pathways. Future regulatory initiatives emphasize safety monitoring and post-market surveillance, affecting product lifecycle management.

Conclusion

The pharmaceutical landscape for busulfan hinges on balancing clinical utility against cost pressures. A nuanced understanding of evolving treatment protocols, patent landscapes, and technological innovations is essential for stakeholders. With sustained growth driven by expanding transplant procedures and formulation enhancements, busulfan remains a valuable asset in oncologic therapeutics, albeit within a highly competitive, cost-conscious environment.

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Key Takeaways

• The busulfan market is expected to grow modestly at 4-6% CAGR, underpinned by increasing transplant volumes and technological advancements.
• Generic formulations dominate due to lower prices, pressuring branded revenues but broadening access.
• Innovations in delivery systems and safety profiles represent strategic opportunities to enhance market share and profitability.
• Competitive threats from emerging conditioning agents and biosimilars require ongoing pipeline innovation and cost management.
• Geographic expansion into emerging markets can offset mature market saturation, provided pricing and regulatory strategies align.

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FAQs

1. How is the market for busulfan projected to evolve over the next five years?
It is expected to grow at a CAGR of approximately 4-6%, driven by increased transplant procedures, formulation improvements, and expanding geographic access, reaching $200-250 million globally by 2028.

2. What factors most influence busulfan’s market viability?
Key factors include clinical necessity in HSCT, regulatory approvals, patent status, competition from biosimilars, and advances in safer, more effective formulations.

3. How do patent expirations impact the busulfan market?
Patents have led to a surge in generic and biosimilar offerings, decreasing prices and market share for branded drugs, while expanding access and volume.

4. Are there any emerging competitors that threaten busulfan’s market dominance?
Yes, agents like treosulfan, targeted therapies, and immunomodulators are under investigation and could diversify or replace busulfan in certain conditioning regimens.

5. What strategic actions can manufacturers pursue to maximize their position?
Innovating in formulation and delivery, establishing robust pharmacokinetic monitoring solutions, and expanding into emerging markets are key strategies to sustain growth.

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Sources:
[1] MarketWatch, “Global Busulfan Market Size, Share & Trends Analysis Report” (2022).
[2] Insights from pharmaceutical industry reports and clinical guidelines (2022).
[3] World Leukemia Association, “Hematologic Malignancies and Transplantation Trends” (2021).