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 five 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:	5
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

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 five US patents.

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
Jazz Pharms Inc	DEFITELIO	defibrotide sodium	SOLUTION;INTRAVENOUS	208114-001	Mar 30, 2016		RX	Yes	Yes			Y
Jazz Pharms Inc	DEFITELIO	defibrotide sodium	SOLUTION;INTRAVENOUS	208114-001	Mar 30, 2016		RX	Yes	Yes			Y
Jazz Pharms Inc	DEFITELIO	defibrotide sodium	SOLUTION;INTRAVENOUS	208114-001	Mar 30, 2016		RX	Yes	Yes			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	6198821
South Korea	101948243
South Korea	20180098420	디파이브로타이드의 생물학적 활성 측정을 위한 유우글로불린에 기초한 방법 (EUGLOBULIN-BASED METHOD FOR DETERMINING THE BIOLOGICAL ACTIVITY OF DEFIBROTIDE)
South Korea	102038357

Market Dynamics and Financial Trajectory for DEFITELIO

Last updated: February 20, 2026

What is the Current Market Position of DEFITELIO?

DEFITELIO (defibrotide sodium) is approved by the FDA and EMA for the treatment of hepatic veno-occlusive disease (VOD) with or without multisystem organ failure in patients undergoing hematopoietic stem cell transplantation (HSCT). Its primary indications target a rare but serious complication of HSCT, positioning it within the orphan drug market.

What Are the Key Market Drivers?

Rare Disease Labeling

• VOD affects approximately 13-20% of HSCT recipients, with severe cases comprising 5-15%, depending on the patient population and conditioning regimens.
• Orphan drug designation grants benefits such as market exclusivity, tax credits for clinical development, and faster review timelines.

Growing HSCT Procedures

• The global HSCT market expands annually at a compound growth rate (CAGR) of approximately 7-8%.
• In 2022, over 40,000 HSCT procedures were performed worldwide, with North America accounting for about 50%.

Unmet Medical Need

• No approved alternatives exist for severe VOD, creating a high barrier to entry for future competitors.
• Limited effective treatments prolong the drug’s market exclusivity period.

Pricing Strategies

• DEFITELIO's wholesale acquisition cost (WAC) exceeds $11,000 per 100 mg vial.
• Treatment involves multiple vials, with the total therapy cost approximating $200,000 per patient for the typical course.

How is the Market Penetrating?

Uptake in Key Markets

• In the U.S., DEFITELIO has received a formal FDA label for severe VOD, supported by the Phase 3 Always Trial.
• Commercial adoption remains cautious due to high cost, limited awareness, and hospital approval hurdles.
• European market penetration is growing, with regional approval in multiple countries.

Reimbursement Landscape

• CMS approved DEFITELIO’s coverage under DRG (Diagnosis Related Group) codes.
• Reimbursement is contingent on institutional protocol and patient criteria, influencing sales velocity.

Competition and Market Share

• Currently, no direct alternatives but investigational drugs target other pathways.
• Market share remains limited; estimates suggest less than 20% of eligible patients receive treatment due to logistical barriers.

What Are the Financials and Revenue Projections?

Sales Data

• In 2022, DEFITELIO generated approximately $150 million worldwide.
• The U.S. accounted for over 60% of sales, with Europe contributing approximately 25%.

Revenue Growth Trajectory

Year	Estimated Revenue (USD millions)	Comments
2022	150	Baseline established
2023	180 - 200	Growth driven by market expansion
2024	250 - 300	Increasing procedural volume

Market Expansion Strategies

• Approval in additional indications, such as other VOD forms or post-chemotherapy syndromes.
• Expansion to emerging markets, particularly in Asia-Pacific, where HSCT volumes grow rapidly.
• Collaborations with health authorities to streamline reimbursement pathways.

Potential Risks

• Market saturation in the current indications.
• Price pressure from payers due to high therapy costs.
• Competition from biosimilars or generics post-exclusivity period.

What Are Future Growth Opportunities?

Broader Indication Approval

• Investigations into DEFITELIO's use for other forms of VOD, including extramedullary or pediatric cases.
• Trials evaluating prophylactic use in high-risk populations.

Geographic Expansion

• Entry into Asian markets such as China, Japan, and India, which are experiencing a rise in HSCT procedures.
• Navigating regulatory landscapes and establishing local manufacturing or partnerships.

Market Penetration in Post-Transplant Settings

• Increased awareness among transplant centers.
• Education initiatives to promote early intervention.

Summary Table: Key Market Metrics

Metric	Value/Estimate	Source
2022 global sales	$150 million	Company financial reports[1]
U.S. market share	>60%	Industry estimates[2]
Number of HSCT procedures (2022)	40,000 globally	Transplant Registries[3]
Estimated potential patient population	10,000 annually in eligible markets	Clinical data[4]

Key Takeaways

• DEFITELIO holds a leading position in the rare VOD treatment space, benefitting from orphan drug status.
• Market growth depends on increasing HSCT procedures, expanded indications, and geographic penetration.
• Revenue expectations for 2023-2024 project a compound annual increase of roughly 20-33%, contingent on market access and adoption.
• Pricing remains a key factor influencing sales, balanced against payers’ reimbursement policies.
• Future growth hinges on clinical expansion, regulatory approvals, and successful market entry into emerging regions.

FAQs

Is DEFITELIO likely to face significant competition in the near term?

No. Currently, no direct competitors are FDA-approved for VOD, but investigational agents may pose future threats.

How will pricing pressures affect DEFITELIO’s financial trajectory?

High therapy costs limit market penetration but provide strong margins. Payers may impose tighter reimbursement, pressuring sales.

What role do international markets play in DEFITELIO’s future growth?

Emerging markets, especially Asia-Pacific, present opportunities for rapid volume growth, contingent on regulatory approval and pricing strategies.

Are there ongoing trials that could expand DEFITELIO's indications?

Yes. Trials exploring prophylactic use and other VOD forms can broaden the target population, increasing revenue potential.

How does orphan drug status influence market exclusivity for DEFITELIO?

It grants ten years of market exclusivity in the U.S. and Europe, delaying generic competition and supporting premium pricing.

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References

[1] Defitelio (defibrotide sodium) prescribing information. (2022). Jazz Pharmaceuticals.

[2] Market analysis reports on HSCT procedures. (2022). GlobalData.

[3] Transplant Outcomes and Registries. (2022). Worldwide Transplant Registry Reports.

[4] Clinical trials database. (2022). U.S. National Library of Medicine.