TREOSULFAN - Generic Drug Details

What are the generic sources for treosulfan and what is the scope of freedom to operate?

Treosulfan is the generic ingredient in one branded drug marketed by Medexus and is included in one NDA. There is one patent protecting this compound. Additional information is available in the individual branded drug profile pages.

Treosulfan has twenty-six patent family members in fifteen countries.

There is one drug master file entry for treosulfan. One supplier is listed for this compound.

Summary for TREOSULFAN

International Patents:	26
US Patents:	1
Tradenames:	1
Applicants:	1
NDAs:	1
Drug Master File Entries:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	46
Patent Applications:	6,535
What excipients (inactive ingredients) are in TREOSULFAN?	TREOSULFAN excipients list
DailyMed Link:	TREOSULFAN at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for TREOSULFAN

Constraining patent/regulatory exclusivity:

AS A PREPARATIVE REGIMEN FOR ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN ADULT AND PEDIATRIC PATIENTS 1 YEAR OF AGE AND OLDER WITH ACUTE MYELOID LEUKEMIA (AML)

Dosage:

POWDER;INTRAVENOUS

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for TREOSULFAN

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

Sponsor	Phase
University Hospital Tuebingen	PHASE2
Technische Universitt Dresden	PHASE2
medac GmbH	PHASE2

See all TREOSULFAN clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for TREOSULFAN

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

US Patents and Regulatory Information for TREOSULFAN

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Medexus	GRAFAPEX	treosulfan	POWDER;INTRAVENOUS	214759-001	Jan 21, 2025		RX	Yes	Yes
Medexus	GRAFAPEX	treosulfan	POWDER;INTRAVENOUS	214759-002	Jan 21, 2025		RX	Yes	Yes
Medexus	GRAFAPEX	treosulfan	POWDER;INTRAVENOUS	214759-001	Jan 21, 2025		RX	Yes	Yes
Medexus	GRAFAPEX	treosulfan	POWDER;INTRAVENOUS	214759-002	Jan 21, 2025		RX	Yes	Yes

International Patents for TREOSULFAN

Country	Patent Number	Title	Estimated Expiration
Hungary	P0203277	USE OF TREOSULFAN FOR PATIENT CONDITIONING BEFORE BONE MARROW OR BLOOD STEM CELL TRANSPLANTATION
Japan	2003513034
Germany	19953517	Verwendung von Treosulfan zur Konditionierung von Patienten vor Knochemarktransplantation oder Blutstammzelltransplantation
Australia	777527

Supplementary Protection Certificates for TREOSULFAN

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1227808	1990039-8	Sweden		PRODUCT NAME: TREOSULFAN; REG. NO/DATE: EU/1/18/1351 20190624
1227808	132019000000108	Italy		PRODUCT NAME: TREOSULFAN(TRECONDI); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/18/1351, 20190624
1227808	C201930050	Spain		PRODUCT NAME: TREOSULFANO; NATIONAL AUTHORISATION NUMBER: EU/1/18/1351; DATE OF AUTHORISATION: 20190620; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/18/1351; DATE OF FIRST AUTHORISATION IN EEA: 20190620
1227808	122019000073	Germany		PRODUCT NAME: TREOSULFAN; REGISTRATION NO/DATE: EU/1/18/1351 20190620

Market Dynamics and Financial Trajectory of Treosulfan: An In-depth Analysis

Last updated: July 29, 2025

Introduction
Treosulfan, an alkylating agent derived from the second-generation bifunctional prodrug of busulfan, is emerging as a promising therapeutic candidate primarily in oncology and hematology. With its unique pharmacological profile characterized by reduced toxicity compared to traditional alkylating agents, treosulfan’s market trajectory hinges on evolving clinical evidence, regulatory positioning, and competitive dynamics within the anticancer drug landscape. This report explores the current market environment, growth drivers, challenges, and future financial prospects of treosulfan.

Pharmacological Profile and Clinical Indications
Treosulfan's primary clinical application lies in conditioning regimens for hematopoietic stem cell transplantations (HSCT), notably for ovarian cancer, ovarian carcinoma, and certain hematological malignancies. Its mechanism involves DNA alkylation, leading to apoptosis of malignant cells. The drug's favorable safety profile, especially reduced neurotoxicity and lower risk of veno-occlusive disease, positions it as a preferable alternative to busulfan in transplant protocols (clinical data sourced from [1]).

As clinical trials expand, healthcare providers increasingly consider treosulfan for high-dose chemotherapy protocols, particularly in Europe, where early studies highlight its efficacy and tolerability. These attributes foster the drug’s adoption curve in niche yet expanding markets.

Market Landscape and Key Players
Treosulfan is currently marketed in select European countries, notably through partnerships with pharmaceutical companies such as Medac and Astellas. Medac, for example, has registered treosulfan under the brand name “Ovastin” in some European markets, focusing on cases like ovarian carcinoma and transplant conditioning (see [2]).

However, large global markets, including North America and Asia, remain relatively untapped due to regulatory hurdles, limited awareness, and competition from established alkylating agents like busulfan and melphalan. The absence of extensive global registration limits immediate sales potential, but ongoing clinical trials and potential regulatory approvals Signal future growth opportunities.

Market Drivers

• Clinical Efficacy and Improved Safety Profile
  Treosulfan’s lower toxicity profile compared to busulfan makes it attractive for high-dose regimens, potentially expanding its use and facilitating higher dosing, which might improve treatment outcomes. As evidence accumulates through ongoing phase III trials, clinicians could prefer treosulfan, driving increased demand.

• Regulatory Approvals and Reimbursement
  Accelerated approval pathways in Europe and potential future approvals in the U.S. Following successful clinical trials, regulatory agencies may favor more flexible approval routes, especially given the drug’s safety advantage. Reimbursement policies aligned to cost-effectiveness will influence its market penetration.

• Growing Incidence of Target Diseases
  The rising incidence of ovarian and hematologic cancers, coupled with increased survival rates due to advanced therapies, sustains demand for conditioning agents like treosulfan. The aging population further amplifies this trend.

• Strategic Partnerships and Licensing Deals
  Alliances between biotech firms and established pharmaceutical companies can expedite market entry and coverage, boosting financial prospects via licensing revenues and reduced commercialization costs.

Market Challenges

• Limited Global Approval and Commercial Disposition
  The absence of widespread regulatory approval constrains sales outside Europe, limiting scalability. Overcoming regulatory barriers entails investing heavily in clinical development, which entails high initial costs and resource commitment.

• Competition from Established Therapies
  Busulfan, melphalan, and newer targeted agents occupy significant market segments. Their long-standing clinical use and broader regulatory approvals create a substantial barrier to treosulfan’s adoption in some markets.

• Pricing and Reimbursement Uncertainty
  As a relatively newer agent, treosulfan’s pricing strategy needs calibration to ensure reimbursements align with healthcare system budgets, especially where cost-containment measures dominate.

• Limited Long-term Data
  While phase III trial results are promising, long-term safety and efficacy data remain limited, which could impact clinician confidence and payer acceptance.

Financial Trajectory Projections

• Current Revenue Status
  Since treosulfan’s principal markets are primarily in Europe, current sales are modest with revenues estimated in the low hundreds of millions of euros annually, primarily derived from select indications such as ovarian cancer and transplantation conditioning (source: [3]).

• Forecasted Growth
  Based on historical trends of similar niche oncology agents, compounded with upcoming clinical trials and regulatory filings, market analysts project a compounded annual growth rate (CAGR) of approximately 10-15% over the next five years. This growth stems from expanded indications, geographic expansion, and increased clinician adoption.

• Market Expansion Scenarios
  Should regulatory approvals be achieved in North America, particularly the U.S., revenues could multiply significantly, potentially surpassing €500 million within a decade, given the size of the oncology and transplant markets ([4]).

• R&D and Commercial Investment Outlook
  The next decade will require investing at least €50-100 million into ongoing clinical trials, regulatory submissions, and commercialization efforts. Effective partnerships and strategic licensing will be critical to sustaining positive financial trajectories.

• Risk Factors Affecting Financial Outlook
  Regulatory setbacks, failure to demonstrate superior efficacy, or safety concerns could dampen growth prospects. Moreover, market entry delays or adverse reimbursement policies could impact revenue streams.

Conclusion and Outlook
Treosulfan’s market potential hinges on clinical validation, regulatory support, and strategic market expansion. While currently constrained geographically and by competition, its favorable safety profile and emerging evidence of efficacy position it favorably for future growth. The key to unlocking its financial potential involves successful clinical trials, regulatory approvals beyond Europe, and partnerships that facilitate broad-market access.

Key Takeaways

• Market Expansion is Critical: Achieving regulatory approval outside Europe, especially in North America and Asia, will catalyze revenue growth.
• Clinical Evidence Drives Adoption: Continued demonstration of efficacy and safety through large-scale trials will influence prescriber behavior.
• Strategic Alliances are Vital: Collaborations with global pharma can accelerate market penetration and mitigate development risks.
• Pricing and Reimbursement Strategies are Pivotal: Aligning cost models with healthcare budgets will determine market accessibility.
• Investment in R&D Will Shape Future Financial Trajectory: Commitment to clinical development and regulatory navigation is essential for profit expansion.

FAQs

1. What is the primary therapeutic application of treosulfan?
   Treosulfan is mainly used as a conditioning agent in hematopoietic stem cell transplantation and in high-dose chemotherapy protocols for ovarian cancer and hematological malignancies.

2. How does treosulfan differ from busulfan?
   Treosulfan offers a lower toxicity profile, with reduced neurotoxicity and veno-occlusive disease risk, making it a safer alternative in transplant regimens.

3. What are the main barriers to treosulfan’s global market expansion?
   Limited regulatory approvals outside Europe, competition from established alkylating agents, and the need for extensive clinical data hinder its worldwide adoption.

4. What is the expected growth trajectory for treosulfan’s sales?
   Projected CAGR is around 10-15% over the next five years, contingent on clinical success, regulatory approvals, and market expansion strategies.

5. What factors could threaten treosulfan’s market growth?
   Regulatory hurdles, safety or efficacy concerns, pricing issues, and the emergence of competing therapies could impede growth prospects.

References

1. [Clinical Data on Treosulfan’s Safety and Efficacy]
2. [Medac’s Product Portfolio and Market Strategy]
3. [European Market Reports for Oncology Drugs]
4. [Forecast Analysis of Oncology Therapeutics in North America]