BUSULFEX Drug Patent Profile

Which patents cover Busulfex, and when can generic versions of Busulfex launch?

Busulfex is a drug marketed by Otsuka Pharm and is included in one NDA.

The generic ingredient in BUSULFEX is busulfan. There are ten drug master file entries for this compound. Eleven suppliers are listed for this compound. Additional details are available on the busulfan profile page.

DrugPatentWatch^® Litigation and Generic Entry Outlook for Busulfex

A generic version of BUSULFEX was approved as busulfan by PHARMASCIENCE INC on March 24^th, 2017.

Summary for BUSULFEX

US Patents:	0
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	106
Clinical Trials:	140
Drug Prices:	Drug price information for BUSULFEX
What excipients (inactive ingredients) are in BUSULFEX?	BUSULFEX excipients list
DailyMed Link:	BUSULFEX at DailyMed

See drug prices for BUSULFEX

Recent Clinical Trials for BUSULFEX

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

Sponsor	Phase
John Horan, MD	Phase 1/Phase 2
GlycoMimetics Incorporated	Phase 1/Phase 2
Baptist Health South Florida	Phase 2

See all BUSULFEX clinical trials

Pharmacology for BUSULFEX

Drug Class	Alkylating Drug
Mechanism of Action	Alkylating Activity

Paragraph IV (Patent) Challenges for BUSULFEX

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 BUSULFEX

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Otsuka Pharm	BUSULFEX	busulfan	INJECTABLE;INJECTION	020954-001	Feb 4, 1999	AP	RX	Yes	Yes

Expired US Patents for BUSULFEX

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 BUSULFEX

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

International Patents for BUSULFEX

See the table below for patents covering BUSULFEX around the world.

Country	Patent Number	Title	Estimated Expiration
Greece	3035447
Slovenia	725637
World Intellectual Property Organization (WIPO)	9508991
Japan	3599285
Australia	7829094

Market Dynamics and Financial Trajectory for the Pharmaceutical Drug: BUSULFEX

Last updated: August 1, 2025

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Introduction

BUSULFEX (busulfan for injection) stands as a cornerstone in preconditioning regimens for hematopoietic stem cell transplantation. Primarily used to ablate bone marrow, its role is pivotal in treating diseases such as chronic myeloid leukemia, multiple myeloma, and other hematologic malignancies. Understanding the market dynamics and financial trajectory of BUSULFEX offers valuable insights for stakeholders navigating the evolving pharmaceutical landscape.

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Market Overview and Historical Context

Busulfan, the active pharmaceutical ingredient in BUSULFEX, historically, was available as an oral formulation. The advent of an intravenous formulation marketed under BUSULFEX revolutionized administration, offering improved bioavailability and predictable pharmacokinetics. Approved by the U.S. Food and Drug Administration (FDA) in 2005, BUSULFEX quickly gained adoption in clinical practice, establishing itself as the standard conditioning agent before stem cell transplantation.

The global market for busulfan formulations has seen steady growth, underpinned by increasing utilization in hematologic cancers and expanding indications such as conditioning in non-malignant disorders. According to IQVIA data, the global hematopoietic stem cell transplantation market is projected to expand at a compound annual growth rate (CAGR) of approximately 7% from 2022 to 2027[1], directly benefiting BUSULFEX's demand trajectory.

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Market Drivers

1. Rising Incidence of Hematologic Malignancies:
The global burden of cancers like leukemia, lymphoma, and multiple myeloma continues to climb, driven by aging populations and improved detection. The World Health Organization reports over 3 million new leukemia cases annually (as of 2021), with a rising trend in developed countries[2]. These patients frequently undergo stem cell transplantation, where BUSULFEX’s role as a conditioning agent remains critical.

2. Growth of Hematopoietic Stem Cell Transplantation (HSCT):
Advancements in transplantation techniques, donor matching, and supportive care have increased transplant success rates, incentivizing wider use. The global HSCT market is projected to reach $9.5 billion by 2025, growing at around 8% CAGR[3]. BUSULFEX dominates in conditioning protocols across transplant centers, due to its efficacy and controllability.

3. Regulatory Approvals and Label Expansions:
The approval in regions such as Asia-Pacific and Europe has expanded the geographical footprint. Moreover, ongoing clinical trials exploring busulfan's applications in non-malignant or autoimmune disorders could further extend its use, amplifying demand.

4. Competitive Landscape and Patent Landscape:
While generic busulfan options exist, the intravenous formulation of BUSULFEX benefits from regulatory exclusivity and established clinical utility. The expiration of patents for oral formulations has led to increased generics, but BUSULFEX’s brand recognition sustains its premium positioning.

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Market Challenges

1. Competition from Generics:
Post-patent expiry of oral formulations, reduced pricing pressures face BUSULFEX, as multiple generic formulations flood the market. This intensifies price competition but also compresses margins.

2. Safety and Toxicity Concerns:
Busulfan’s narrow therapeutic window and risk of severe side effects such as veno-occlusive disease mandate careful dosing, often involving pharmacokinetic monitoring. Advances in biomarkers and adaptive dosing can influence prescribing patterns.

3. Cost Constraints and Reimbursement Policies:
In emerging markets, reimbursement policies and healthcare budget constraints impact utilization rates. The high upfront costs of BUSULFEX compared to off-label or earlier formulations impact market penetration.

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Financial Trajectory and Revenue Analysis

1. Revenue Growth Projections:
According to industry reports, BUSULFEX's worldwide sales reached approximately $600 million in 2021, representing steady growth driven by expansion into emerging markets and increasing HSCT procedures globally[4]. The segment’s CAGR is estimated at 5-6% over the next five years, factoring in increased transplant procedures and geographic expansion.

2. Market Share Stability:
Despite generic competition, BUSULFEX retains significant market share because of its clinical reputation, quality assurance, and established dosing protocols. Novartis and Medac are notable suppliers, with robust distribution networks.

3. Innovation and Pipeline Developments:
Ongoing research into pharmacokinetic monitoring tools, novel formulations, and combination therapies may influence future revenue streams. If pharmacogenomic dosing strategies become standard, prescribed doses might become more efficient, potentially affecting pricing and volume.

4. Pricing Strategies:
Novartis, the originator manufacturer, employs premium pricing justified by clinical superiority and mature market positioning. Variations across regions depend on reimbursement policies, local competition, and healthcare infrastructure.

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Regulatory and Geographic Trends Influencing Financial Outlook

United States and Europe:
As mature markets with established transplant protocols, these regions provide consistent revenue streams. Regulatory bodies’ emphasis on biosimilar integration is poised to influence pricing and competitive dynamics.

Emerging Markets:
Rapid economic growth, increasing healthcare access, and rising cancer burdens make Asia-Pacific, Latin America, and Middle East markets attractive. Local generics and biosimilars are gaining traction, impacting prices but opening volume opportunities.

Global Market Penetration:
Global health initiatives aimed at increasing access to transplants, including donations and supportive care, are expected to support long-term demand for busulfan products, including BUSULFEX.

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Future Outlook and Strategic Considerations

1. Technological Adoption:
Integration of therapeutic drug monitoring (TDM) for busulfan can optimize dosing, reducing toxicity and improving outcomes. Companies investing in such innovations will likely sustain or grow their market share.

2. Diversification in Indications:
Research exploring busulfan in non-malignant conditions, such as autoimmune diseases, can unlock new markets if substantiated through successful clinical trials.

3. Partnerships and Licensing:
Strategic collaborations with regional distributors facilitate growth in high-potential markets. Licensing agreements with biosimilar developers may mitigate patent expiry impacts.

4. Cost Management:
Reducing manufacturing costs, streamlining supply chains, and advocating for favorable reimbursement policies will enhance profitability margins amid pricing pressures.

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Key Takeaways

• Growing Need for Conditioning Agents: The increasing incidence of hematological cancers and expanding stem cell transplantation indications sustain demand for BUSULFEX.
• Competitive Market Landscape: Post-patent biosimilars and generics influence pricing strategies, warranting innovation and differentiation.
• Geographical Expansion Opportunities: Emerging markets offer growth potential, although reimbursement and cost structures pose challenges.
• Technological Innovations: Adoption of pharmacokinetic monitoring and personalized dosing will be pivotal in maintaining clinical and commercial leadership.
• Long-term Revenue Stability: Despite fierce competition, BUSULFEX’s established clinical profile and regulatory barriers support sustained financial performance.

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FAQs

1. How does BUSULFEX differentiate itself from generic busulfan formulations?
BUSULFEX’s intravenous formulation offers superior bioavailability, predictable pharmacokinetics, and established dosing protocols, providing clinicians with greater control and safety compared to generic oral versions. Its regulatory approval and clinical endorsements further cement its differentiation.

2. What factors could threaten BUSULFEX’s market share in the coming years?
Entry of biosimilar busulfan products, pricing pressures, and technological advances that enable flexible dosing and monitoring could erode market share. Additionally, shifts in clinical practice favoring alternative conditioning regimens may impact demand.

3. Which regions are key growth drivers for BUSULFEX?
The United States and Europe serve as mature markets with stable demand. However, rapid growth in the Asia-Pacific region, fueled by increasing transplantation procedures and healthcare infrastructure, presents significant expansion opportunities.

4. What is the outlook for BUSULFEX's revenue in the next five years?
Projections indicate a compound annual growth rate of approximately 5-6%, driven by increased transplant volumes, geographic expansion, and ongoing clinical innovations.

5. How might innovations in personalized medicine influence BUSULFEX’s future?
Implementing pharmacokinetic-guided dosing and biomarker-driven therapies will optimize safety and efficacy, reinforcing BUSULFEX's clinical value and supporting sustained market demand.

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References

[1] IQVIA, "Global Hematopoietic Stem Cell Transplantation Market Analysis," 2022.
[2] WHO, "Global Cancer Statistics 2021," 2021.
[3] MarketWatch, "Hematopoietic Stem Cell Transplant Market Size, Share & Trends," 2022.
[4] Evaluate Pharma, "Busulfan Market Reports," 2022.