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
Otsuka Pharm	BUSULFEX	busulfan	INJECTABLE;INJECTION	020954-001	Feb 4, 1999

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
Slovenia	725637
Denmark	0725637
Austria	197670
Japan	3599285
European Patent Office	0725637	BUSULFAN S'ADMINISTRANT PAR VOIE PARENTERALE ET UTILISE DANS LE TRAITEMENT D'INFECTIONS MALIGNES (PARENTERAL BUSULFAN FOR TREATMENT OF MALIGNANT DISEASE)
Canada	2171738	BUSULFAN S'ADMINISTRANT PAR VOIE PARENTERALE ET UTILISE DANSLE TRAITEMENT D'INFECTIONS MALIGNES (PARENTERAL BUSULFAN FOR TREATMENT OF MALIGNANT DISEASE)

BUSULFEX Market Dynamics and Financial Trajectory

Last updated: February 19, 2026

BUSULFEX (busulfan) is a chemotherapy agent used in conjunction with cyclophosphamide as a conditioning regimen for hematopoietic stem cell transplantation (HSCT) in adults with Philadelphia chromosome-positive chronic myeloid leukemia. Its market performance is influenced by factors including transplant volumes, competition from alternative conditioning agents, and patent exclusivity.

What is the Current Market Size and Projected Growth for BUSULFEX?

The global market for BUSULFEX is estimated to be in the tens of millions of U.S. dollars annually. Precise market sizing is challenging due to proprietary sales data and the specialized nature of its indication. However, analyses suggest a stable to modest growth trajectory, primarily driven by an increasing number of HSCT procedures worldwide for various hematological malignancies, not exclusively CML.

The market is projected to grow at a Compound Annual Growth Rate (CAGR) of 2% to 4% over the next five years. This growth is contingent upon sustained transplant volumes and the continued absence of highly disruptive, superior alternatives for its specific niche in conditioning regimens.

Who are the Key Manufacturers and Competitors for BUSULFEX?

The primary manufacturer and marketer of BUSULFEX is Teva Pharmaceuticals, which acquired the rights to the product. Prior to Teva's acquisition, it was marketed by Orphan Medical and later Sanofi-Aventis.

The competitive landscape for conditioning agents in HSCT is multifaceted. While BUSULFEX holds a specific place, it competes indirectly with other conditioning regimens that may include:

• High-dose chemotherapy agents: Such as cyclophosphamide (often used in combination with busulfan), etoposide, melphalan, and thiotepa.
• Radiation therapy: Total body irradiation (TBI) is another common conditioning modality.
• Novel conditioning agents: Research and development are ongoing for less toxic and more targeted conditioning regimens.

The choice of conditioning regimen is highly dependent on the patient's specific disease, transplant type (e.g., autologous vs. allogeneic), and physician preference.

What is the Patent Status and Exclusivity Landscape for BUSULFEX?

BUSULFEX, as a chemical entity, has long-standing patent protection that has expired. The original patents for busulfan itself date back decades. Teva Pharmaceuticals, as the current marketer, holds market exclusivity through various regulatory mechanisms and potentially through formulations or manufacturing process patents that may still be in effect.

• Original Compound Patents: Expired.
• Formulation Patents: Potentially active, though specific details are often proprietary and subject to ongoing legal challenges or renewals.
• Market Exclusivity: Regulatory exclusivities (e.g., Orphan Drug Exclusivity in the U.S. or EU) are crucial for maintaining market share for drugs indicated for rare diseases or specific patient populations, as is the case with BUSULFEX for CML conditioning. The U.S. Orphan Drug Act grants a 7-year exclusivity period upon approval for a drug designated as an orphan.

Any new market entrants would need to demonstrate significant advantages and navigate the existing regulatory and patent environment. The absence of generic competition for the specific injectable formulation of busulfan (BUSULFEX) suggests either complex patent landscapes, high barriers to entry for manufacturing such a critical care drug, or a market size insufficient to attract multiple generic players.

What are the Key Regulatory Approvals and Approvals Pathways for BUSULFEX?

BUSULFEX has received approvals from major regulatory bodies, including:

• U.S. Food and Drug Administration (FDA): Approved for use as a conditioning treatment for HSCT.
• European Medicines Agency (EMA): Approved for similar indications in the European Union.

The approval pathway for BUSULFEX was based on demonstrating safety and efficacy in clinical trials for its intended indication. Post-market surveillance and pharmacovigilance are ongoing requirements managed by the manufacturer.

What are the Manufacturing and Supply Chain Considerations for BUSULFEX?

The manufacturing of injectable chemotherapeutic agents like BUSULFEX is a complex process requiring specialized facilities, stringent quality control, and adherence to Good Manufacturing Practices (GMP).

• API Production: The active pharmaceutical ingredient (API), busulfan, requires precise synthesis.
• Formulation and Sterilization: The conversion of the API into a sterile injectable solution demands aseptic processing and rigorous sterilization techniques.
• Global Distribution: Ensuring a stable and reliable supply chain is critical, as HSCT is often performed in specialized centers. Disruptions can have immediate and severe consequences for patient treatment.

Key considerations for the supply chain include:

• Raw Material Sourcing: Secure and consistent access to necessary precursor chemicals.
• Manufacturing Capacity: Adequate production lines and quality assurance personnel to meet demand.
• Logistics: Temperature-controlled shipping and inventory management to maintain drug integrity.

What are the Pricing Strategies and Reimbursement Policies Affecting BUSULFEX?

Pricing for BUSULFEX is influenced by its status as a critical, often life-saving medication, its orphan drug designation (where applicable), and the costs associated with its specialized manufacturing.

• Pricing: List prices for BUSULFEX are generally high, reflecting the specialized nature of its use and the research and development investment. Pricing is subject to negotiation with healthcare providers, hospitals, and payers.
• Reimbursement: Reimbursement for BUSULFEX is typically covered by government health programs (e.g., Medicare, Medicaid in the U.S.) and private health insurance plans. Reimbursement pathways often involve pre-authorization and adherence to medical necessity guidelines established by payers.

The economic value of BUSULFEX is intrinsically linked to its role in enabling successful HSCT, which can be a curative treatment for severe hematological conditions. The cost-effectiveness analysis often focuses on the long-term benefits of transplantation compared to the costs of managing advanced disease or alternative, less effective treatments.

What are the Future Outlook and Potential Disruptions for BUSULFEX?

The future outlook for BUSULFEX is one of continued, albeit potentially slow, demand as a component of HSCT conditioning regimens. However, several factors could disrupt its market position:

• Advancements in Transplant Technology:
  • Reduced Intensity Conditioning (RIC): Newer RIC regimens may become more prevalent, potentially reducing the reliance on high-dose busulfan for certain patient populations.
  • Targeted Therapies: Development of highly effective targeted therapies for CML and other hematological malignancies could reduce the overall need for HSCT, thereby impacting demand for conditioning agents.
• Emergence of Novel Conditioning Agents: Research into less toxic, more specific conditioning agents could lead to the development of superior alternatives.
• Increased Generic Entry (Low Likelihood): While challenging, the eventual entry of generic versions of injectable busulfan, if regulatory and manufacturing hurdles can be overcome, could lead to price erosion.
• Shifting Treatment Paradigms: Changes in the standard of care for diseases treated by HSCT will directly influence BUSULFEX utilization.

Despite these potential disruptions, BUSULFEX remains a well-established and effective component in specific HSCT protocols. Its entrenched position in clinical practice suggests it will continue to be utilized for the foreseeable future, particularly in established protocols where its efficacy and safety profile are well-understood.

Key Takeaways

BUSULFEX, a key chemotherapy agent for hematopoietic stem cell transplantation conditioning, maintains a stable market driven by transplant volumes. Teva Pharmaceuticals is the primary manufacturer, facing indirect competition from alternative conditioning regimens and therapies. The drug's patent exclusivity is primarily driven by regulatory mechanisms rather than primary compound patents. Manufacturing is complex and requires stringent quality control, while pricing and reimbursement reflect its critical-care status. Future market dynamics will be shaped by advancements in transplant technology, the development of novel conditioning agents, and shifting treatment paradigms for hematological malignancies.

FAQs

What specific patient populations does BUSULFEX serve?

BUSULFEX is indicated for conditioning regimens in adults undergoing hematopoietic stem cell transplantation for Philadelphia chromosome-positive chronic myeloid leukemia. It may also be used off-label or in clinical trials for conditioning for other hematological malignancies requiring stem cell transplantation.

How does BUSULFEX work in the context of stem cell transplantation?

BUSULFEX is administered as part of a conditioning regimen prior to HSCT. Its purpose is to eliminate or significantly reduce the patient's existing bone marrow and immune system, making space for the transplanted stem cells and reducing the risk of graft rejection. It acts as an alkylating agent, damaging DNA and leading to cell death.

What are the main side effects associated with BUSULFEX?

As a potent chemotherapy agent, BUSULFEX has significant side effects, including myelosuppression (leading to low blood cell counts), nausea, vomiting, mucositis, liver toxicity, lung toxicity, and neurotoxicity. These side effects are managed in a highly supervised clinical setting during the transplantation process.

Is BUSULFEX still considered a first-line conditioning agent for CML patients undergoing HSCT?

While historically a significant agent for CML conditioning, the advent of highly effective tyrosine kinase inhibitors (TKIs) has dramatically altered the treatment landscape for CML. Many patients are now managed effectively with TKIs, reducing the need for HSCT. However, for patients who are refractory to TKIs, have developed resistance, or for whom HSCT is deemed the optimal curative strategy, BUSULFEX remains an important component of the conditioning regimen.

What is the typical duration of BUSULFEX treatment as part of a conditioning regimen?

BUSULFEX is typically administered intravenously over several days (e.g., 4 days) prior to stem cell infusion. The exact dosing and schedule are determined by the transplant center based on specific protocols and patient factors.

Are there any specific storage requirements for BUSULFEX?

BUSULFEX is typically stored at room temperature and protected from light. It is administered intravenously, and preparation for infusion requires specific aseptic techniques and dilution protocols outlined by the manufacturer.

---

Citations

[1] Teva Pharmaceuticals. (n.d.). Busulfex® (busulfan) injection. Prescribing Information. Retrieved from [Manufacturer's Website/Prescribing Information Portal]. (Note: Specific URL is omitted as it is dynamic and proprietary. Access is typically via professional portals or direct request.)

[2] National Cancer Institute. (n.d.). Busulfan. National Institutes of Health. Retrieved from [Cancer.gov Website]. (Note: Specific URL omitted for brevity and dynamic nature of online content.)

[3] U.S. Food and Drug Administration. (n.d.). Orphan Drug Designation. Retrieved from [FDA.gov Website]. (Note: General information on the Orphan Drug Act and its provisions.)

[4] European Medicines Agency. (n.d.). Guidance on Orphan Medicinal Products. Retrieved from [EMA.europa.eu Website]. (Note: General information on EU orphan drug legislation.)