DEFIBROTIDE SODIUM - Generic Drug Details

What are the generic drug sources for defibrotide sodium and what is the scope of freedom to operate?

Defibrotide sodium is the generic ingredient in one branded drug marketed by Jazz Pharms Inc and is included in one NDA. There are three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Defibrotide sodium has twenty-five patent family members in sixteen countries.

One supplier is listed for this compound.

Summary for DEFIBROTIDE SODIUM

International Patents:	25
US Patents:	3
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	9
Clinical Trials:	1
What excipients (inactive ingredients) are in DEFIBROTIDE SODIUM?	DEFIBROTIDE SODIUM excipients list
DailyMed Link:	DEFIBROTIDE SODIUM at DailyMed

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

Constraining patent/regulatory exclusivity:

11,236,328

Dosage:

SOLUTION;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 DEFIBROTIDE SODIUM

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

Sponsor	Phase
University of California, San Francisco	Phase 2
Jazz Pharmaceuticals	Phase 2

See all DEFIBROTIDE SODIUM clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for DEFIBROTIDE SODIUM

B01AX	Other antithrombotic agents
B01A	ANTITHROMBOTIC AGENTS
B01	ANTITHROMBOTIC AGENTS
B	Blood and blood forming organs

Paragraph IV (Patent) Challenges for DEFIBROTIDE SODIUM

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
DEFITELIO	Injection	defibrotide sodium	200 mg/2.5 mL	208114	1	2024-12-27

US Patents and Regulatory Information for DEFIBROTIDE SODIUM

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Jazz Pharms Inc	DEFITELIO	defibrotide sodium	SOLUTION;INTRAVENOUS	208114-001	Mar 30, 2016		RX	Yes	Yes	11,236,328
Jazz Pharms Inc	DEFITELIO	defibrotide sodium	SOLUTION;INTRAVENOUS	208114-001	Mar 30, 2016		RX	Yes	Yes	11,085,043		Y
Jazz Pharms Inc	DEFITELIO	defibrotide sodium	SOLUTION;INTRAVENOUS	208114-001	Mar 30, 2016		RX	Yes	Yes	11,746,348		Y

International Patents for DEFIBROTIDE SODIUM

Country	Patent Number	Title	Estimated Expiration
Japan	2015521477	デフィブロチドの生物学的活性を決定する為の、ユーグロブリンに基づく方法
Singapore	11201408481U
Mexico	2014016114
South Korea	20150044877
Singapore	11201408481U	EUGLOBULIN-BASED METHOD FOR DETERMINING THE BIOLOGICAL ACTIVITY OF DEFIBROTIDE
China	104619857

Market Dynamics and Financial Trajectory for Defibrotide Sodium

Last updated: February 14, 2026

Overview: Defibrotide sodium is a poorly soluble oligonucleotide derived from porcine intestinal mucosa. It is approved primarily for the treatment of hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), particularly in stem cell transplant patients. Market access and financial prospects hinge on approval status, clinical data, and emerging applications.

Regulatory Status and Market Approval

• United States: Approved by the FDA in 2016 under the brand name Defitelio for severe VOD post-HSCT in adult and pediatric patients.
• European Union: Approved since 2013 under the same indication.
• Japan: Approved in 2014.
• Other Markets: Several countries follow EMA and FDA approvals; however, market penetration remains limited largely to North America, Europe, and Japan.

Market Size and Revenue Estimates

• Global VOD Market: Estimated at approximately $500 million in 2022.
• Defibrotide Contribution: Accounts for roughly 90% of VOD treatment sales. Vendors like Jazz Pharmaceuticals (acquired rights via accelerated pathways) dominate this space.
• Sales Trajectory (2022–2027): Projected Compound Annual Growth Rate (CAGR) of approximately 10-12%. Growth driven by increasing transplant procedures and expanded use in other indications.

Year	Estimated Global Sales	Key Drivers
2022	$450 million	Established usage, limited competition
2023	$495 million	Market penetration in additional territories, new patient populations
2024	$550 million	Expanded indications including off-label uses; new formulations
2025	$610 million	Growing transplant volume; potential off-label recognition
2027	$750 million	Broader clinical acceptance, increased transplant rates

Market Drivers

• Increase in Hematopoietic Stem Cell Transplants (HSCT): Growing global transplant rates, with an estimated 50,000 procedures annually.
• Regulatory Expansions: Recent approvals for additional indications such as severe hepatic VOD in children.
• Limited Competition: Few approved alternatives; other potential treatments are in various stages of development.
• Pricing Dynamics: High prices per treatment course (~$50,000–$100,000), supported by limited competition.

Market Challenges

• Pricing and Reimbursement: Payers increasingly scrutinize cost-effectiveness. Reimbursement varies significantly by country.
• Access Barriers: Limited approval outside North America, Europe, and Japan stifles expansion.
• Clinical Limitations: Efficacy primarily demonstrated in HSCT-related VOD. Unclear benefits for off-label uses.

Emerging Opportunities

• New Formulations: Development of oral formulations or shorter infusion protocols.
• Indication Expansion: Research into treatment of other microvascular or coagulopathy-related conditions.
• Orphan Drug Designation: Maintains market exclusivity in many regions, protecting revenue streams.

Financial Outlook and Investment Considerations

• For companies with rights, defibrotide sodium remains a cash-generating therapy with limited patent expiration risk.
• Future growth prospects depend on regulatory approvals for expanded indications and geographic expansion.
• R&D investments focus on novel formulations and downstream indications.

Policy and Market Trends

• Emphasis on personalized medicine tailoring treatments to high-risk transplant patients.
• Increasing reimbursement pressures influencing pricing strategies.
• Market consolidation, with Jazz Pharmaceuticals maintaining dominant market share.

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Key Takeaways

• Defibrotide sodium's revenue growth is driven by transplant volume increases and expanded indications.
• Sales are likely to grow at double-digit CAGR through 2027, contingent on regulatory and market access expansion.
• Competition remains limited, supporting pricing power, though reimbursement pathways pose challenge.
• Innovation in formulations and broader clinical applications could open additional revenue streams.

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FAQs

1. What are the main indications for defibrotide sodium? Principal use in severe hepatic VOD following HSCT. Emerging research explores applications in other microvascular disorders.

2. How does defibrotide sodium compare to potential competitors? Currently, few approved alternatives exist; investigational drugs are in early or late clinical phases but have not yet achieved market entry.

3. What are the primary risks affecting its market growth? Regulatory delays, reimbursement restrictions, limited geographic approval, and off-label use restrictions.

4. How sensitive is the market to changes in transplant volumes? Highly sensitive; growth in HSCT procedures directly correlates with increased demand for defibrotide sodium.

5. What are the prospects for off-label use? Potential exists, but regulatory and reimbursement hurdles limit commercial expansion outside approved indications.

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Citations

1. FDA. Defitelio (defibrotide sodium) prescribing information. 2016.
2. European Medicines Agency. EMA approval details for defibrotide. 2013.
3. MarketWatch. Global Hematopoietic Stem Cell Transplant Market & VOD Treatment Market. 2022.
4. Jazz Pharmaceuticals. Annual Report 2022.
5. EvaluatePharma. Pharmaceutical revenue projections, 2022.