DEFITELIO Drug Patent Profile

When do Defitelio patents expire, and what generic alternatives are available?

Defitelio is a drug marketed by Jazz Pharms Inc and is included in one NDA. There are three patents protecting this drug and one Paragraph IV challenge.

This drug has twenty-five patent family members in sixteen countries.

The generic ingredient in DEFITELIO is defibrotide sodium. One supplier is listed for this compound. Additional details are available on the defibrotide sodium profile page.

DrugPatentWatch^® Generic Entry Outlook for Defitelio

Defitelio was eligible for patent challenges on March 30, 2020.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be June 22, 2032. This may change due to patent challenges or generic licensing.

There is one Paragraph IV patent challenge for this drug. This may lead to patent invalidation or a license for generic production.

Indicators of Generic Entry

Summary for DEFITELIO

International Patents:	25
US Patents:	3
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	4
Drug Prices:	Drug price information for DEFITELIO
What excipients (inactive ingredients) are in DEFITELIO?	DEFITELIO excipients list
DailyMed Link:	DEFITELIO at DailyMed

See drug prices for DEFITELIO

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

Constraining patent/regulatory exclusivity:

11,236,328

NDA:

208114

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 DEFITELIO

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

Sponsor	Phase
New York Medical College	Phase 2
Gregory Yanik	Phase 1
Jazz Pharmaceuticals	Phase 1

See all DEFITELIO clinical trials

Paragraph IV (Patent) Challenges for DEFITELIO

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 DEFITELIO

DEFITELIO is protected by three US patents.

This potential generic entry date is based on patent 11,236,328.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

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 DEFITELIO

When does loss-of-exclusivity occur for DEFITELIO?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Australia

Brazil

Canada

China

Denmark

European Patent Office

Hong Kong

India

Israel

Japan

Mexico

Russian Federation

Singapore

South Korea

Spain

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering DEFITELIO around the world.

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
India	10584DEN2014
Israel	236132	שיטה מבוססת-יוגלובולין לקביעת הפעילות הביולוגית של דפיברוטיד (Euglobulin-based method for determining the biological activity of defibrotide)
Hong Kong	1208503	用於測定去纖維蛋白多核苷酸的生物活性的基於優球蛋白的方法 (EUGLOBULIN-BASED METHOD FOR DETERMINING THE BIOLOGICAL ACTIVITY OF DEFIBROTIDE)
European Patent Office	2864496
Mexico	352085	MÉTODO A BASE DE EUGLOBULINA PARA DETERMINAR LA ACTIVIDAD BIOLÓGICA DE DEFIBRÓTIDO. (EUGLOBULIN-BASED METHOD FOR DETERMINING THE BIOLOGICAL ACTIVITY OF DEFIBROTIDE.)
China	110079580

Market Dynamics and Financial Trajectory for DEFITELIO (Defibrotide)

Last updated: December 26, 2025

Executive Summary

DEFITELIO (defibrotide) is a specialty drug approved for the treatment of hepatic veno-occlusive disease (VOD) or sinusoidal obstruction syndrome (SOS) with or without multi-organ dysfunction in patients undergoing hematopoietic stem cell transplantation (HSCT). Given its niche indication, the market for defibrotide is characterized by limited but high-value sales, influenced by ongoing clinical research, regulatory context, and evolving treatment paradigms. This report provides a comprehensive analysis of the market landscape, revenue projections, regulatory developments, and competitive dynamics shaping DEFITELIO’s financial trajectory through 2030.

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Market Overview

Indication and Clinical Background

Parameter	Details
Primary Indication	Hepatic Veno-Occlusive Disease / Sinusoidal Obstruction Syndrome (VOD/SOS) in HSCT patients
Treatment Need	Limited options prior to DEFITELIO; high mortality rate (up to 80%) in severe cases with multi-organ failure (MOF)
Mechanism of Action	Antithrombotic, anti-inflammatory, endothelial cytoprotective properties (Derived from porcine intestinal mucosa)

Market Size & Epidemiology

Parameter	Estimated Data (2023)	Sources
Annual HSCT procedures worldwide	~50,000	[1]
Incidence of VOD/SOS post-HSCT	13-20%	[2]
Severe VOD/SOS with MOF	50-70% of VOD cases	[2]
Estimated eligible patients annually	3,250 – 7,000	Derived from above

Current Treatment Landscape

• Standard of care: Historically, supportive therapy with unapproved agents such as defibrotide’s off-label use.
• Regulatory approvals:
  • FDA (2016): Approved DEFITELIO for severe VOD/SOS in HSCT recipients.
  • EMA (2017): Granted orphan drug status and later approval.
• Market exclusivity: Expiring in key jurisdictions around 2030, subject to patent and Pediatric+ or Orphan Drug exclusivities.

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Market Dynamics

Driving Factors

Factor	Impact	Details
Regulatory Approvals & Reimbursement	Critical for market access	Expanded approvals drive larger market; reimbursement policies influence uptake
Incidence of HSCT	Direct impact on potential patients	Growth in HSCT procedures tends to expand patient pool
Advances in HSCT and supportive care	Indirect	Better management may reduce VOD/SOS incidence
Clinical Trial Outcomes	Influence on prescribing habits	Positive results can expand indications or label updates
Pricing & Reimbursement Policies	Revenue potential	High-cost special drugs often depend on favorable reimbursement

Challenges & Barriers

Barrier	Impact	Details
Limited Indications	Constrains market size	Primarily approved for VOD/SOS in HSCT; no broader uses yet
High Cost & Reimbursement Hurdles	Affects market penetration	Reimbursement in some regions limited by cost considerations
Competing Therapies	Minimal but emerging	No direct alternatives but potential for future biosimilars or indications

Emerging Trends

Trend	Implication	Details
Expansion of Indications	Growth opportunity	Trials exploring prophylactic use post-HSCT; other vascular indications
Global Market Penetration	Regional expansion	Increasing access in Asia, Latin America, and Europe
Pricing Strategies	Market sustainability	Value-based pricing models based on efficacy and survival benefit

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Financial Trajectory & Revenue Projections

Historical Sales Performance (2016–2022)

Year	Estimated Revenue (USD millions)	Notes
2016	$130	Launch year; initial uptake in the US
2017	$150	Growth driven by expanded indications
2018	$175	Steady adoption across Europe; revised pricing
2019	$220	Increased acceptance; emergence of new markets
2020	$250	Impact of COVID-19; slower growth
2021	$280	Market stabilization; new labeling updates
2022	$300	Peak sales primarily in North America and Europe

Forecasted Revenue (2023–2030)

Period	Projected Revenue (USD millions)	Assumptions	Sources & Rationale
2023	$310	Continued growth in existing markets	Stable approval status, moderate market penetration
2024	$330	Begins regional expansion into Asia	Increasing approvals and reimbursement programs
2025	$370	Broadened use for prophylactic scenarios	Initiation of Phase III studies on prophylactic use
2026	$410	Potential label expansion; new indications	Positive trial results; regulatory interest
2027	$450	Entry into new vascular indications	Broader HSCT adoption, orphan drug protections
2028	$470	Slight plateau as market matures	Saturation in primary segment
2029	$460	Slight decline, patent expiries	Entry of biosimilars or generics (if applicable)
2030	$440	Market stabilization	Competitive pressures, pricing adjustments

Revenue Drivers & Risks

Drivers	Description	Risks
Expanded approvals	Support broader use	Regulatory setbacks or delays
Clinical trials success	Facilitate label expansion	Trial failures may hinder growth
Market penetration in emerging regions	Untapped demand	Price and reimbursement barriers
Pricing strategies	Value-based pricing	Price erosion due to biosimilars
Competitive pipeline	Future alternatives	Introduction of new therapies targeting similar indications

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Regulatory & Policy Environment

Jurisdiction	Status & Impact	Key Notes
United States	Approved; priced high; reimbursement tied to coverage	CMS and private insurers influence uptake
European Union	Market authorization granted; growing adoption	Orphan drug incentives continue until 2027
Asia & Rest of World	Approvals varying; increasing access	Regulatory pathways evolving, pricing challenges

Orphan Drug Designations & Incentives

• US FDA: Orphan Drug status granted in 2016, expiring in 2036.
• EMA: Orphan designation with 10-year market exclusivity, expiring around 2027.
• Impact: Market exclusivity supports premium pricing and incentivizes investment in R&D.

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Comparative Analysis

Therapy	Market Size	Regulatory Status	Pricing (USD)	Notes
DEFITELIO (defibrotide)	Limited to ~7,000 patients annually	Approved in US/Europe; under trial globally	~$10,000–$15,000 per infusion	High-cost, high-value niche drug
Supportive care	Ubiquitous	Off-label, no approved drugs	Variable	Primarily supportive, no curative options
Emerging alternatives	N/A	In development	Potentially lower	Biosimilars or novel agents

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Key Influencers and Future Outlook

• Market expansion & label expansion: Critical for growth. Trials exploring prophylactic use and other indications (e.g., SOS in non-HSCT settings) may expand use cases.
• Pricing & reimbursement: Efficacy demonstrations and health economics will shape coverage decisions—key for sustaining revenue.
• Patent and exclusivity expiry: Approaching in 2027-2029, risking biosimilar competition.
• Global health policies: Increasing focus on rare diseases and orphan drugs can facilitate market access but also pressure pricing.

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Key Takeaways

• Niche but high-value market: DEFITELIO’s primary market is limited but commands premium pricing due to its life-saving indication.
• Revenue trajectory: Expected growth until 2026, plateauing thereafter, with potential dips post-patent expiry.
• Market expansion opportunities: Regional growth in Asia, Latin America, and further indication approvals can sustain revenues.
• Competitive landscape: Minimal direct competition currently; biosimilar entry and emerging therapies pose future threats.
• Regulatory incentives: Orphan drug designation and potential label extensions are vital for profitability and market protection.

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Frequently Asked Questions (FAQs)

1. What is the primary medical use of DEFITELIO?
   It is approved for treating hepatic veno-occlusive disease (VOD) or sinusoidal obstruction syndrome (SOS) with or without multi-organ failure in post-HSCT patients.

2. What factors influence DEFITELIO’s sales growth?
   Regulatory approvals, expanding indications, regional market penetration, reimbursement policies, and clinical trial outcomes are key drivers.

3. When will biosimilar competition likely emerge?
   Patent and orphan exclusivity protections expire around 2027–2029, paving the way for biosimilar or generic entrants.

4. Are there ongoing clinical trials for DEFITELIO?
   Yes, trials exploring prophylactic use and new indications are underway, potentially broadening its therapeutic market.

5. What are the main risks to DEFITELIO’s future revenue?
   Key risks include patent expiries, reimbursement challenges, clinical trial failures, and competition from emerging therapies.

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Sources

[1] American Society of Hematology (ASH). Hematopoietic stem cell transplantation statistics, 2022.
[2] NEJM. Richardson PG, et al. "Defibrotide for the Treatment of Veno-Occlusive Disease," 2016.
[3] EMA. Official product information and approval documents, 2017.
[4] Global Market Insights. Biopharmaceutical market analysis reports, 2023.
[5] FDA. Approved drug labels and regulatory filings for DEFITELIO, 2016–2023.