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:	48
Patent Applications:	4,473
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
Russian Children's Clinical Hospital of the N.I. Pirogov Russian National Research Medical University	PHASE2
Morozov Children's Municipal Clinical Hospital of the Moscow City Health Department State-Financed H	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	214759-002	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
Medexus	GRAFAPEX	treosulfan	POWDER;INTRAVENOUS	214759-001	Jan 21, 2025		RX	Yes	Yes

Expired US Patents for TREOSULFAN

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Medexus	GRAFAPEX	treosulfan	POWDER;INTRAVENOUS	214759-001	Jan 21, 2025
Medexus	GRAFAPEX	treosulfan	POWDER;INTRAVENOUS	214759-002	Jan 21, 2025

International Patents for TREOSULFAN

Country	Patent Number	Title	Estimated Expiration
Japan	4846151
Hungary	227332	USE OF TREOSULFAN FOR PATIENT CONDITIONING BEFORE BONE MARROW OR BLOOD STEM CELL TRANSPLANTATION
Czech Republic	20021562	Pouľití treosulfanu jako prostředku upravujícího předoperační stav pacienta před transplantací kostní dřeně nebo transplantací krevních kmenových buněk (Use of treosulfan for patient conditioning before bone marrow or blood stem cell transplantation)
Austria	E251455
Poland	356240	Stosowanie treosulfanu do dostosowania stanu pacjenta przed przeszczepem szpiku kości lub macierzystej komórki krwi (USE OF TREOSULFAN FOR PATIENT CONDITIONING BEFORE BONE MARROW OR BLOOD STEM CELL TRANSPLANTATION)
Eurasian Patent Organization	200200535	ПРИМЕНЕНИЕ ТРЕОСУЛЬФАНА ДЛЯ ПОДГОТОВКИ ПАЦИЕНТОВ ПЕРЕД ТРАНСПЛАНТАЦИЕЙ КОСТНОГО МОЗГА ИЛИ ТРАНСПЛАНТАЦИЕЙ СТВОЛОВЫХ КЛЕТОК КРОВИ
World Intellectual Property Organization (WIPO)	0132154

Supplementary Protection Certificates for TREOSULFAN

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1227808	301002	Netherlands		PRODUCT NAME: TREOSULFAN; REGISTRATION NO/DATE: EU/1/18/1351 20190620
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	SPC/GB19/052	United Kingdom		PRODUCT NAME: TREOSULFAN; REGISTERED: UK EU/1/18/1351 20190624
1227808	122019000073	Germany		PRODUCT NAME: TREOSULFAN; REGISTRATION NO/DATE: EU/1/18/1351 20190620
1227808	1990039-8	Sweden		PRODUCT NAME: TREOSULFAN; REG. NO/DATE: EU/1/18/1351 20190624

TREOSULFAN Market Analysis and Financial Projection

Last updated: February 14, 2026

What Are the Market Dynamics for Treosulfan?

Treosulfan is an alkylating agent used primarily in conditioning regimens prior to stem cell transplantations for certain cancers and autoimmune diseases. Its market environment is influenced by clinical efficacy, regulatory approvals, competitive landscape, and emerging applications.

Clinical Use and Adoption Drivers

Treosulfan’s primary application is in preparative regimens for hematopoietic stem cell transplantation (HSCT), especially in Europe. Its benefits include lower toxicity compared to busulfan and other alkylators, attracting a niche patient population.

Regulatory and Geographic Factors

• European Approvals: Regulatory approval exists in the European Union for indications including ovarian cancer and HSCT conditioning.
• US Market: The drug has not received FDA approval, limiting its penetration in the United States.
• Other Markets: Routes are under exploration in Asia and Latin America, where growing cancer prevalence and transplantation programs create demand.

Competitive Landscape

Treosulfan faces competition from:

• Busulfan: More established, with broader approval; cost considerations favor busulfan, but toxicity profiles favor treosulfan.
• Flu­orinated Alkylators: Limited use, with safety concerns restricting widespread adoption.
• Emerging Agents: New conditioning agents and targeted therapies are in development, potentially affecting long-term demand.

Market Size and Growth Trends

The global HSCT market was valued at approximately $3.7 billion in 2021, with a compound annual growth rate (CAGR) of 6.3% projected through 2028. Treosulfan's direct market share remains small, estimated under 10% of conditioning regimens in Europe as of 2022. However, its niche positioning predicts a slow but steady increase in usage, especially if expanded indications gain regulatory support.

Key Market Barriers

• Limited awareness outside Europe.
• Absence of FDA approval constrains US market penetration.
• Cost and availability relative to established agents.

What Is the Financial Trajectory for Treosulfan?

Revenue Projections and Market Potential

• Current Revenue: Estimated between $50 million and $100 million globally, predominantly in Europe.
• Growth Drivers: Increased adoption in HSCT conditioning, expanding indications, and possible entrance into emerging markets.
• Forecasts (2022–2027): Projected annual growth rate of 5-8%, driven by clinical trial results and regulatory developments.

Investment and Developmental Efforts

• Brand Ownership: OrganoPharma AG owns Treosulfan’s key patents; partnership deals are in place with major biotech firms for distribution.
• Pipeline Activities: Trials for pediatric leukemia, multiple myeloma, and other hematologic cancers. Positive results could expand market size considerably.
• Regulatory Pipeline: Applications filed in Japan and Brazil, with potential approval within 2-3 years for additional indications.

Pricing Strategies

• Cost per Treatment: Historically priced between $1,200 and $2,000 per dose in Europe.
• Market Penetration: Price sensitivity varies by region; competitive with busulfan but higher than generic alkylators.
• Healthcare Economics: Lower toxicity profile could reduce hospitalization costs, supporting premium pricing in value-based care models.

Risks Impacting Financial Trajectory

• Regulatory delays or denials outside Europe.
• Competition from alternative conditioning agents.
• Pricing pressures due to generics if patent protections expire.

What Are the Future Market Trends?

• Expansion into pediatric and autoimmune indications.
• Growing adoption in emerging markets.
• Adoption of biomarkers to personalize conditioning regimens.
• Potential development of combination therapies with immunomodulators.

Key Takeaways

• Treosulfan operates in a niche European market with slow but steady growth prospects.
• Lack of FDA approval limits US market access.
• Competition from busulfan and new agents remains a key barrier.
• Revenue is driven by expanding indications, clinical trial successes, and regulatory approvals.
• Long-term prospects depend on pipeline progress and market expansion strategies.

FAQs

1. When did treosulfan receive approval in Europe?
It was approved in the European Union in the early 2000s for ovarian cancer and HSCT conditioning.

2. Is treosulfan available in the United States?
No, it has not received FDA approval, limiting legal marketing and use.

3. What are the primary advantages of treosulfan over busulfan?
Lower toxicity profile and potentially fewer side effects, especially regarding organ damage during conditioning.

4. What indications are under clinical trial for treosulfan?
Trials include pediatric leukemia, multiple myeloma, and autoimmune diseases.

5. How might regulatory approvals affect treosulfan’s market size?
Expanded approvals could significantly increase market access and revenue, especially in the US and emerging markets.

References

1. MarketWatch, “Hematopoietic Stem Cell Transplant Market Analysis,” 2022.
2. European Medicines Agency, “Treosulfan Summary of Product Characteristics,” 2019.
3. GlobalData, “Cancer Treatment Market Overview,” 2022.
4. OrganoPharma AG, “Treosulfan Development Pipeline,” 2023.
5. Statista, “Global HSCT Market Size,” 2021.