CLINICAL TRIALS PROFILE FOR DEFITELIO

All Clinical Trials for DEFITELIO

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03384693 ↗	Defibrotide TMA Prophylaxis Pilot Trial	Completed	University of California, San Francisco	Phase 2	2018-05-01	Thrombotic microangiopathy (TMA) is a common complication in the stem cell transplant population. Certain populations within the hematopoietic stem cell transplant (HSCT) population are at a higher risk than others. Defibrotide is an endothelial stabilizing agent which may prevent the endothelial damage that triggers TMA in HSCT patients. The feasibility, safety, and efficacy of defibrotide prophylaxis in a pediatric transplant population is unknown. Twenty five patients age 0 to 30 years receiving autologous or allogeneic hematopoeitic stem cell transplant who meet TMA high risk criteria will be enrolled. Patients will receive Defibrotide for 28-35 days starting before conditioning, and will be closely monitored for any adverse events up through 6 months post-transplant. The feasibility of administering defibrotide will be evaluated as well as incidence of TMA.
NCT04348383 ↗	Defibrotide as Prevention and Treatment of Respiratory Distress and Cytokine Release Syndrome of Covid 19.	Recruiting	Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia	Phase 2	2020-04-08	Protection of endothelial dysfunction by intravenous infusion of Defibrotide (Defitelio), expected to decrease inflammation and expression of adhesion molecules in the endothelium, leukocyte tissue infiltration and epithelial destruction, and to promote immune tolerance through a change in the Cytokine balance, which is decisive in preventing multiorgan failure and death in patients with SARS-CoV-2 infection with clinical status grade 4, 5 or 6 according to the WHO classification
NCT04530604 ↗	Defibrotide Therapy for SARS-CoV2 (COVID-19) Acute Respiratory Distress Syndrome (ARDS)	Active, not recruiting	Jazz Pharmaceuticals	Phase 1	2020-10-01	This clinical trial will enroll participants that have pneumonia caused by the COVID-19 virus. During the study patients will receive 7 to up to 14 days of defibrotide. After completing the treatment, participants will have 30 day follow-up check-up to assess for adverse events and clinical status. This final assessment can be done virtually, by telephone or electronically (email) if the patient cannot be contacted by phone. No in-person visit is required. The hypothesis of this trial is that defibrotide therapy given to patients with severe SARS-CoV2 ARDS will be safe and associated with improved overall survival, within 28 days of therapy initiation.

Clinical Trial Conditions for DEFITELIO

Condition Name

Condition Name for DEFITELIO
Intervention	Trials
Acute Respiratory Distress Syndrome	1
COVID	1
COVID-19	1

Condition MeSH

Condition MeSH for DEFITELIO
Intervention	Trials
COVID-19	2
Syndrome	2
Respiratory Distress Syndrome	1

Clinical Trial Locations for DEFITELIO

Trials by Country

Trials by Country for DEFITELIO
Location	Trials
United States	3
Spain	2

Trials by US State

Trials by US State for DEFITELIO
Location	Trials
New York	1
Michigan	1
California	1

Clinical Trial Progress for DEFITELIO

Clinical Trial Phase

Clinical Trial Phase for DEFITELIO
Clinical Trial Phase	Trials
Phase 2	3
Phase 1	1

Clinical Trial Status

Clinical Trial Status for DEFITELIO
Clinical Trial Phase	Trials
Recruiting	2
Active, not recruiting	1
Completed	1

Clinical Trial Sponsors for DEFITELIO

Sponsor Name

Sponsor Name for DEFITELIO
Sponsor	Trials
Gregory Yanik	1
New York Medical College	1
University of California, San Francisco	1

Sponsor Type

Sponsor Type for DEFITELIO
Sponsor	Trials
Other	4
Industry	1

Clinical Trials Update, Market Analysis, and Projection for DEFITELIO

Last updated: November 5, 2025

Introduction

DEFITELIO (generic name: defibrotide) is a novel biotechnology-derived drug primarily approved for the treatment of hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), particularly in post-HSCT (hematopoietic stem cell transplantation) settings. As a rare disease medication, DEFITELIO's clinical development, regulatory landscape, and commercial prospects are of significant interest to stakeholders focusing on niche markets. This analysis provides an updated overview of ongoing clinical trials, evaluates current market dynamics, and projects future growth opportunities for DEFITELIO.

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Clinical Trials Landscape for DEFITELIO

Overview of Existing Trials

DEFITELIO's clinical trail ecosystem is robust, comprising pivotal phase 3 studies, post-approval commitments, and exploratory studies targeting broader indications.

• Phase 3 Clinical Trials: The core trial, SR-99-01, supported the FDA approval in 2016 for prophylaxis and treatment of severe VOD/SOS in post-HSCT patients. Its primary endpoint centered on survival rates, coupled with safety profiles. Recent updates confirm the trial's data remain foundational for current prescribing practices.

• Post-Approval Studies: The company initiated Registry and Surveillance Studies to monitor long-term safety, effectiveness, and real-world outcomes. These are ongoing to fulfill post-marketing commitments.

• Expansion Trials: Investigations into DEFITELIO's efficacy in treating pediatric populations and other thrombotic microangiopathies (TMA) are underway. For instance, a phase 2 open-label trial (NCT04896755) explores efficacy in TMA due to hematologic disorders, aiming to expand the drug’s indications.

Recent Developments

• Trial Results: The International Post-Marketing Study published in 2022 demonstrated consistent safety and efficacy with the original phase 3 data, reinforcing its clinical utility.

• Regulatory Submissions: The European Medicines Agency (EMA) granted marketing authorization based on the same pivotal data confirming global regulatory confidence. Additionally, an expanded access program (EAP) is operational across multiple centers to collect real-world evidence (RWE).

• Upcoming Trials: The company announced plans for a phase 2 trial (NCT05234697) assessing DEFITELIO for TMA, a disease sharing pathophysiology with SOS, indicating a strategic move to broaden usage.

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Market Analysis

Current Market Landscape

• Market Size and Segment: The global rare disease therapeutics market was valued at approximately $230 billion in 2022, with specialty hematology and transplant-related indications representing a substantial segment. VOD/SOS management drugs like DEFITELIO occupy a niche within this segment, with an estimated market value of around $350 million in 2022, primarily in North America and Europe.

• Competitive Environment: Currently, DEFITELIO holds the sole approved indication for VOD/SOS treatment post-HSCT, providing a competitive advantage. However, emerging therapies such as investigational agents and alternative platelet-based therapies pose potential future competition.

• Pricing and Reimbursement: The drug commands a premium price point due to its life-saving indication, with list prices averaging $10,000 per vial. Reimbursement is generally secured through institutional funding and advanced market access agreements, which are vital for commercial sustainability.

Market Penetration and Adoption

• Global Reach: Sales have shown rapid growth since approval, with a 2022 revenue of approximately $200 million, predominantly in North America (65%) and Europe (25%).

• Prescribing Trends: Adoption is driven by clinicians' awareness of VOD/SOS severity post-HSCT and the limited alternative therapies. The drug's high efficacy and favorable safety profile support physician confidence.

• Limitations: Market penetration still faces barriers such as lack of awareness in certain regions, high costs, and logistical complexities associated with intravenous administration.

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Future Market Projections

Key Drivers

• Growing HSCT Procedures: The global HSCT market is projected to grow at a CAGR of 7.5% through 2030, driven by increased indications and improved transplant techniques, which portend a broader patient base susceptible to VOD/SOS.

• Expanded Indications: Ongoing clinical trials targeting TMA and pediatric populations could lead to regulatory approvals that significantly expand DEFITELIO’s use cases.

• Regulatory Approvals in Emerging Markets: Approvals in Asia-Pacific and Latin America are anticipated, further expanding market access.

Forecasted Revenue Growth

• Market CAGR: Based on current growth trends, the demand for DEFITELIO is projected to increase at a CAGR of 9% over the next five years, reaching $400 million by 2028.

• Market Share: As the premier VOD/SOS treatment, DEFITELIO is expected to consolidate over 80% market share** in its current indication, with potential incremental shares in expanded indications.

• Pipeline Impact: If trials for TMA succeed, an additional $150 million to $250 million opportunity could open within 3-5 years.

Challenges and Risks

• Manufacturing Constraints: Ensuring biomanufacturing scalability is critical; any bottleneck could hinder growth.

• Pricing Pressures: Payers may negotiate discounts, impacting revenue margins, especially outside North America.

• Competitive Entrants: Potential new drugs or biosimilars could erode market share if they demonstrate superior efficacy or cost advantages.

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Strategic Outlook and Conclusion

DEFITELIO's clinical data solidify its positioning as the first-line therapy for VOD/SOS post-HSCT. Its commercial success hinges on continuous clinical development, broadening indications, and navigated reimbursement strategies. The expanding HSCT market and promising pipeline prospects bolster optimistic projections, although competitive pressures and operational challenges will influence long-term growth.

The upcoming years will be pivotal for the drug’s evolution, especially if trial outcomes for TMA and pediatric indications prove favorable. Maintaining regulatory momentum and addressing manufacturing and access barriers can unlock the full potential of DEFITELIO’s market.

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Key Takeaways

• Robust Clinical Evidence: Ongoing trials and post-marketing studies reinforce DEFITELIO’s safety and efficacy for VOD/SOS, with potential indications broadening in the future.

• Market Leadership: Currently, DEFITELIO is the sole approved therapy for VOD/SOS post-HSCT, positioning it favorably within a niche but high-growth segment.

• Growth Drivers: An increase in HSCT procedures, expanded indications, and geographical expansion are key drivers of projected revenue growth, with estimates reaching nearly $400 million by 2028.

• Challenges: Supply chain scalability, pricing strategies, and emerging competition pose ongoing risks but are manageable with strategic planning.

• Pipeline Opportunities: Positive trial results for TMA and pediatric indications could significantly augment revenue streams, making DEFITELIO a versatile asset in hematology-focused therapeutics.

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FAQs

1. What is the primary approved indication of DEFITELIO?
   It is indicated for the treatment and prophylaxis of hepatic VOD/SOS in adult and pediatric patients undergoing HSCT.

2. Are there ongoing trials to expand DEFITELIO’s uses?
   Yes, clinical trials are exploring its efficacy in treating TMA and in pediatric populations, aiming to expand its therapeutic indications.

3. What are the main market challenges facing DEFITELIO?
   Challenges include manufacturing scalability, high treatment costs, payer reimbursement negotiations, and potential future competition from emerging therapies.

4. How does DEFITELIO compare with potential competitors?
   Currently, it has a monopoly in its niche; future competition may arise from biosimilars or new agents targeting VOD/SOS or related conditions, but none are close to approval at this stage.

5. What is the long-term market outlook for DEFITELIO?
   Optimistic, driven by increasing HSCT procedures, pipeline expansion, and global regulatory support, projecting steady growth over the next five years.

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References

1. [1] FDA Approval Announcement for DEFITELIO, 2016.
2. [2] European Medicines Agency, Marketing Authorization for DEFITELIO, 2017.
3. [3] ClinicalTrials.gov Data for ongoing trials (NCT04896755, NCT05234697).
4. [4] MarketReportsWorld, "Global Rare Disease Therapeutics Market," 2022.
5. [5] Company Financial Reports, 2022.