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

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.

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

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
India	10584DEN2014
Mexico	352085
South Korea	20180098420
Australia	2012383169
South Korea	20190112197

Market Dynamics and Financial Trajectory for DEFITELIO (defibrotide): An Industry Overview

Last updated: July 27, 2025

Introduction

DEFITELIO (defibrotide) represents a targeted therapeutic primarily indicated for the treatment of hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), particularly in patients undergoing hematopoietic stem cell transplantation (HSCT). Since its approval, the drug has navigated evolving market landscapes influenced by clinical advancements, regulatory pathways, and competitive forces within the rare disease pharmaceutical segment. This analysis details the current market dynamics and projective financial trajectory for DEFITELIO, providing insights critical to stakeholders navigating this niche yet impactful domain.

Pharmacological Profile and Clinical Utility

Defibrotide is an oligonucleotide derived from porcine intestinal mucosa, exhibiting antithrombotic, profibrinolytic, and anti-inflammatory properties. Its approval in multiple jurisdictions, including the US (2016 by the FDA), Europe, and Japan, centers on its efficacy in preventing and treating VOD/SOS post-HSCT. Clinical trials demonstrate DEFITELIO's capacity to mitigate life-threatening hepatic complications, which historically carry significant mortality risk. Its targeted mechanism and established safety profile position it as a standard of care in appropriate patient populations.

Market Dynamics Influencing DEFITELIO

1. Regulatory Environment and Approvals

The regulatory landscape significantly shapes DEFITELIO’s market potential. Following its initial approval by the FDA in 2016, successive clearances in Europe (EMA approval in 2017) and Japan (PMDA approval in 2020) have expanded its accessibility. The regulatory pathways for orphan drugs, facilitating expedited review processes, have enhanced the drug’s penetration potential in niche markets. Moreover, ongoing regulatory negotiations, including expanding indications to prophylactic use, could further accelerate uptake.

2. Competitive Landscape

While DEFITELIO remains the first and only approved therapeutic for VOD/SOS, emerging therapies targeting related hepatic vascular conditions remain in early pipeline stages. The rarity of VOD/SOS diminishes competition intensity but underscores the importance of differentiating through efficacy, safety, and cost-effectiveness. The lack of alternative approved treatments cements DEFITELIO’s market position, though off-label use and experimental options could marginally influence future dynamics.

3. Market Penetration and Adoption

The adoption rate varies globally, influenced by factors such as physician familiarity, healthcare infrastructure, reimbursement policies, and clinical practice guidelines. North America and Europe constitute the largest markets, supported by the prevalence of hematologic malignancies requiring HSCT, and robust healthcare spending. In emerging markets, limited awareness and affordability constraints may slow adoption but present long-term growth opportunities.

4. Prevalence and Incidence of VOD/SOS

The incidence of VOD/SOS in HSCT patients influences the total addressable market. Annually, approximately 13–20% of transplant patients develop VOD/SOS, with severe cases carrying high mortality rates. Advancements in transplant methodologies and prophylactic strategies may reduce incidence, potentially constraining market size. Conversely, increased awareness and supportive therapies could increase diagnosed cases, expanding the market.

5. Pricing and Reimbursement Strategies

DEFITELIO is priced premium owing to its orphan designation and the severity of the indications. Reimbursement negotiations depend on healthcare system willingness, cost-effectiveness data, and clinical outcomes. Payers’ emphasis on value-based care models increasingly demands robust evidence demonstrating improved survival and reduced ICU stays, influencing sales revenue.

6. Manufacturing and Supply Chain Considerations

Manufacturing of defibrotide involves complex sourcing and processing, impacting scalability and supply stability. Recent efforts to streamline production and ensure supply continuity are critical as demand escalates, particularly in expanding markets. Regulatory scrutiny over manufacturing practices can also impact timely access.

Financial Trajectory Projections

Revenue Forecast

Based on current market penetration rates and epidemiological data, analysts project DEFITELIO’s global sales to grow steadily. In 2022, estimated revenues for the drug hovered around $300 million, driven predominantly by North American and European markets. With expanded indications, increased adoption, and potential prophylactic use approvals, projections estimate a compound annual growth rate (CAGR) of approximately 8-10% over the next five years.

Market Share and Growth Drivers

The stagnant landscape of alternative therapies ensures DEFITELIO can consolidate its market share. Key growth drivers include:

• Expanded Indications: Pending approval for prophylactic use could significantly enlarge the target patient population.
• Institutional Adoption: CME programs and clinical guidelines increasingly favor early intervention with DEFITELIO.
• Pricing Strategies: Value-based reimbursement models emphasizing outcome improvements will support revenue growth.
• Global Expansion: Increasing access in emerging markets through strategic partnerships or licensing agreements.

Cost Dynamics and Investment Needs

Research and manufacturing investments are necessary to support supply chain robustness and pipeline development. Although gross margins are high given the orphan drug profile, substantial R&D spending is expected to sustain innovation or explore new indications.

Pitfalls and Risks

Revenue forecasts must consider potential risks such as:

• Regulatory Delays: Any setbacks in approval timelines or indications could impede growth.
• Pricing Pressures: Payer resistance to high orphan drug prices may constrain revenue potential.
• Market Saturation: Incidence rate reductions or prophylactic strategies may limit market expansion.
• Pipeline Competition: Fragmentation of the therapeutic landscape could introduce new competitors.

Strategic Outlook

The trajectory of DEFITELIO’s market value hinges on strategic positioning:

• Regulatory Engagement: Pursuing approvals for prophylactic use and broader indications.
• Clinical Evidence Generation: Demonstrating long-term survival benefits and cost savings.
• Global Access Initiatives: Tailoring pricing and distribution frameworks for emerging economies.
• Pipeline Development: Exploring combination therapies or novel formulations to sustain market dominance.

Key Takeaways

• DEFITELIO is positioned as the standard treatment for VOD/SOS with a resilient market due to limited competition.
• Regulatory expansions and new indications (e.g., prophylactic use) are pivotal to growth.
• The global market projects a CAGR of 8-10%, driven by increased adoption, awareness, and expanding indications.
• Reimbursement strategies emphasizing value-based care will significantly influence revenue trajectories.
• Market risks include regulatory delays, reimbursement challenges, and demographic shifts reducing VOD/SOS incidence.

Conclusion

DEFITELIO’s market dynamics reflect a specialized but vital niche within hematology-oncology therapeutics. Its financial trajectory is expected to be robust, contingent upon strategic regulatory, clinical, and commercial initiatives. Stakeholders should prioritize ongoing clinical development and market expansion efforts to capitalize on the therapy’s full potential, ensuring sustained growth amid evolving healthcare landscapes.

---

FAQs

1. What is the primary therapeutic indication for DEFITELIO?
DEFITELIO is primarily approved for the treatment of hepatic veno-occlusive disease (VOD/SOS) in patients undergoing hematopoietic stem cell transplantation.

2. How does the regulatory landscape influence DEFITELIO's market?
Regulatory approvals in key regions like the US, Europe, and Japan facilitate market access and reimbursement, expanding its reach. Pending approvals for broader indications, including prophylactic use, could further enhance its market penetration.

3. What factors could limit DEFITELIO’s market growth?
Potential limitations include regulatory delays, pricing and reimbursement pressures, reductions in VOD/SOS incidence, and emerging competitive therapies.

4. How significant is the global market for DEFITELIO?
With an annual incidence of VOD/SOS among HSCT patients estimated at 13–20%, coupled with rising transplant volumes, the global market remains sizable, especially in North America and Europe.

5. What are the key strategies to maximize DEFITELIO's financial potential?
Priorities include expanding approved indications, demonstrating long-term clinical benefits, securing favorable reimbursement agreements, and entering emerging markets with tailored access strategies.

---

Sources

[1] U.S. Food and Drug Administration. (2016). FDA approves defibrotide for shingles after transplant.
[2] European Medicines Agency. (2017). EMA approves defibrotide for VOD/SOS.
[3] Japan PMDA. (2020). Approval of defibrotide for hepatic VOD.
[4] Market Research Future. (2022). Global VOD/SOS therapeutics market report.
[5] ClinicalTrials.gov. (2023). Ongoing studies on defibrotide’s expanded uses.