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:	42
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
Assistance Publique - Hôpitaux de Paris	Phase 3
Heinrich-Heine University, Duesseldorf	Phase 1/Phase 2
Koordinierungszentrum für Klinische Studien - Duesseldorf	Phase 1/Phase 2

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	214759-001	Jan 21, 2025		RX	Yes	Yes	7,199,162
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

International Patents for treosulfan

Country	Patent Number	Title	Estimated Expiration
Australia	1391801	Use of treosulfan for patient conditioning before bone marrow or blood stem cell transplantation
Hungary	227332	USE OF TREOSULFAN FOR PATIENT CONDITIONING BEFORE BONE MARROW OR BLOOD STEM CELL TRANSPLANTATION
European Patent Office	1227808	UTILISATION DE TREOSULFANE POUR LE CONDITIONNEMENT DE PATIENTS AVANT UNE GREFFE DE MOELLE OSSEUSE OU UNE GREFFE DE CELLULES SANGUINES SOUCHES (USE OF TREOSULFAN FOR PATIENT CONDITIONING BEFORE BONE MARROW OR BLOOD STEM CELL TRANSPLANTATION)
Eurasian Patent Organization	004543	ПРИМЕНЕНИЕ ТРЕОСУЛЬФАНА ДЛЯ ПОДГОТОВКИ ПАЦИЕНТОВ ПЕРЕД ТРАНСПЛАНТАЦИЕЙ КОСТНОГО МОЗГА ИЛИ ТРАНСПЛАНТАЦИЕЙ СТВОЛОВЫХ КЛЕТОК КРОВИ (USE OF TREOSULFAN FOR PATIENT CONDITIONING BEFORE BONE MARROW OR BLOOD STEM CELL TRANSPLANTATION)

Supplementary Protection Certificates for treosulfan

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
1227808	122019000073	Germany		PRODUCT NAME: TREOSULFAN; REGISTRATION NO/DATE: EU/1/18/1351 20190620
1227808	301002	Netherlands		PRODUCT NAME: TREOSULFAN; REGISTRATION NO/DATE: EU/1/18/1351 20190620
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	132019000000108	Italy		PRODUCT NAME: TREOSULFAN(TRECONDI); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/18/1351, 20190624

Treosulfan Market Analysis and Financial Projection

Last updated: March 23, 2025

Treosulfan, an alkylating agent used in conditioning regimens for allogeneic hematopoietic stem cell transplantation (alloHSCT) in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), has become a focal point in hematology-oncology markets following its FDA approval in January 2025[9][13]. Its financial and market trajectory reflects a mix of clinical promise, strategic commercialization, and economic sustainability.

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Market Dynamics

Clinical Superiority and Adoption

• Treosulfan demonstrated a 33% reduction in mortality risk compared to busulfan in a Phase 3 trial, with 2-year event-free survival rates of 64.0% vs. 50.4% for busulfan[9][11]. This clinical edge has positioned it as a preferred conditioning regimen.
• Market uptake projections were initially underestimated by the manufacturer, but independent analyses (e.g., CADTH) revised adoption to 40% in Year 1, 60% in Year 2, and 80% in Year 3 post-approval, reflecting clinician confidence in its efficacy[1].

Cost-Effectiveness and Sustainability

• A cost-effectiveness analysis showed treosulfan saves €41,784 per patient compared to busulfan while improving quality-adjusted life-years (+0.96)[4]. Over five years, this could reduce healthcare system expenses by €179,174[4].
• CADTH emphasized that treosulfan should not exceed the cost of busulfan to ensure reimbursement parity, limiting pricing flexibility despite its clinical advantages[12].

Regulatory and Competitive Landscape

• Treosulfan holds Orphan Drug Designation in the U.S., granting 7.5 years of market exclusivity[13]. This shields it from generic competition in the near term.
• While it targets AML/MDS initially, analysts suggest expanded indications (e.g., other transplant uses) could unlock higher revenue potential[10].

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Financial Trajectory

Revenue Projections

• Medexus anticipates peak U.S. revenue exceeding $100 million annually within five years of commercialization[2][10][14]. Early forecasts from Leede Financial model $83–96 million by FY33–34, contingent on pricing (~$8,500–9,500 per procedure) and adoption rates[10].
• In Canada, unit demand for Trecondyv® grew 55% YoY as of December 2024, signaling strong initial traction[14].

Medexus’s Financial Position

• Q3 Fiscal 2025 Results: Revenue of $30.0 million, with *Adjusted EBITDA of $5.8 million (21% margin)[14]. Debt was reduced to $40.9 million**, improving liquidity[14].
• Investments in treosulfan’s U.S. launch totaled $0.4–0.5 million in early 2025, with plans to scale spending ahead of the commercial rollout[8].

Risks and Challenges

• Regulatory Delays: Past setbacks, including a 2022 FDA rejection due to manufacturing concerns, highlight dependency on smooth approvals[5][7].
• Pricing Pressure: Analysts note busulfan’s generic status limits treosulfan’s pricing power unless clinical differentiation is emphasized[10][12].

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Strategic Outlook

• Commercial Launch: Targeted for 1H 2025 in the U.S., with product availability by April 2025[13][14]. Revenue impact is expected by early 2026[8].
• Portfolio Synergies: Medexus aims to leverage treosulfan’s success to strengthen its rare disease portfolio, supported by 21.5% ROE projections and steady gross margins (~53–54%)[2][14].

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Key Takeaway: Treosulfan’s trajectory hinges on balancing clinical adoption with cost containment. While its $100M revenue target is ambitious, Medexus’s strategic investments and treosulfan’s orphan drug status provide a credible pathway to disrupt the alloHSCT conditioning market.

References

1. https://www.ncbi.nlm.nih.gov/books/NBK605551/
2. https://www.equityreads.com/p/medexus-pharmaceuticals-inc-tsx-mdp
3. https://www.cantechletter.com/2024/06/is-mdp-stock-a-buy-june-2024/
4. https://journals.aboutscience.eu/index.php/grhta/article/view/2412
5. https://financialpost.com/globe-newswire/medexus-provides-update-on-treosulfan-nda-resubmission-and-provides-business-update
6. https://www.newsfilecorp.com/release/169672/Medexus-Highlights-Data-on-Trecondyv-Treosulfan-Presented-at-MDS-2023
7. https://www.medexus.com/en_US/news-media/press-releases/detail/102/medexus-pharmaceuticals-reports-financial-and-operational
8. https://www.stocktitan.net/news/MEDXF/medexus-announces-strong-fiscal-q2-2025-x2pe9r0eipdv.html
9. https://healthtree.org/blood-cancer/community/articles/fda-approves-treosulfan-for-aml-mds
10. https://www.streetwisereports.com/article/2024/12/06/fda-decision-on-drug-for-bone-marrow-due-early-2025.html
11. https://www.medexus.com/en_US/investors/news-events/press-releases/detail/181/medexus-and-ontarios-provincial-health-services
12. https://www.cda-amc.ca/sites/default/files/DRR/2024/PC0324%20Trecondyv%20-%20Draft%20CADTH%20Recommendation.pdf
13. https://www.medexus.com/en_US/investors/news-events/press-releases/detail/176/medexus-announces-fda-approval-of-grafapex-treosulfan-for
14. https://www.medexus.com/en_US/investors/news-events/press-releases/detail/182/medexus-announces-strong-fiscal-q3-2025-results