~ Buy the DEFITELIO (defibrotide sodium) Drug Profile, 2024 PDF Report in the Report Store ~

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.
NCT04530604 ↗	Defibrotide Therapy for SARS-CoV2 (COVID-19) Acute Respiratory Distress Syndrome (ARDS)	Active, not recruiting	Gregory Yanik	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
COVID	1
COVID-19	1
COVID19	1
Sars-CoV2	1

Condition MeSH

Condition MeSH for defitelio
Intervention	Trials
COVID-19	2
Syndrome	2
Respiratory Distress Syndrome	1
Acute Lung Injury	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
Completed	1
Active, not recruiting	1

Clinical Trial Sponsors for defitelio

Sponsor Name

Sponsor Name for defitelio
Sponsor	Trials
Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia	1
Jazz Pharmaceuticals	1
Gregory Yanik	1

Sponsor Type

Sponsor Type for defitelio
Sponsor	Trials
Other	4
Industry	1

Defitelio Market Analysis and Financial Projection

Defitelio (Defibrotide Sodium): Clinical Trials, Market Analysis, and Projections

Introduction to Defitelio

Defitelio, also known as defibrotide sodium, is a medication approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of hepatic veno-occlusive disease (VOD) with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT)[4][5].

Clinical Trials Overview

The efficacy of Defitelio was established through three clinical trials involving 528 patients with hepatic VOD and multi-organ dysfunction after HSCT. These trials included two prospective clinical trials and one expanded access study.

Survival Rates

The primary efficacy outcome was the survival rate at 100 days after HSCT. The trials showed that 38% to 45% of patients treated with Defitelio were alive at Day +100, compared to 21% to 31% for patients receiving only supportive care or other interventions[1][4][5].

Demographic Analysis

The drug demonstrated similar efficacy across different demographic subgroups:

• Sex: Defitelio worked equally well in male and female patients.
• Race: Although the majority of participants were White, the small number of participants from other races made it difficult to determine differences.
• Age: The drug was effective in patients both younger and older than 16 years, but there were too few patients aged 65 or older to draw conclusions[1].

Trial Design

The trials were open-label, with patients receiving Defitelio intravenously at a dose of 6.25 mg/kg every 6 hours for a median of 21 days (range 1-83 days). The treatment was continued until the resolution of VOD or up to 60 days[1][4].

Market Analysis

Current Market Performance

Defitelio is part of Jazz Pharmaceuticals' portfolio, and while the company's financial reports do not provide detailed sales figures specifically for Defitelio, the overall performance of Jazz Pharmaceuticals' oncology products has been strong. The company's total revenues have seen a slight increase, driven by growth in various product lines, although Defitelio's contribution is not explicitly highlighted[2].

Market Need and Competition

Hepatic VOD is a severe and life-threatening condition with a high mortality rate, often exceeding 75%. The approval of Defitelio fills a critical need for a treatment option in this area, as it significantly improves survival rates compared to supportive care alone[5].

Pricing and Reimbursement

The pricing of Defitelio is not publicly disclosed in the sources provided, but given its orphan drug status and the critical nature of the condition it treats, it is likely to be a high-cost medication. Reimbursement policies vary by region and insurance provider, but the drug's approval under expedited programs suggests a favorable regulatory environment for its use[4].

Projections and Future Outlook

Market Growth Potential

Given the severe nature of hepatic VOD and the lack of alternative treatments, Defitelio has significant market growth potential. The drug's ability to improve survival rates and reduce mortality makes it a valuable option for patients undergoing HSCT.

Regulatory Environment

Defitelio was granted Priority Review by the FDA, indicating a favorable regulatory environment for its use. This expedited approval process reflects the drug's importance in treating a serious condition with limited treatment options[4].

Potential Expansion

Future growth could be driven by expanded indications or broader use in related conditions. However, any expansion would require additional clinical trials to establish efficacy and safety in new patient populations.

Adverse Reactions and Safety Profile

Defitelio has a notable safety profile, with major potential adverse reactions including hemorrhage and hypersensitivity reactions. Common adverse reactions include hypotension, diarrhea, vomiting, nausea, and epistaxis. The use of Defitelio is contraindicated in patients being treated concurrently with anticoagulants or fibrinolytic therapies due to its profibrinolytic activity[4].

Key Takeaways

• Improved Survival Rates: Defitelio significantly improves survival rates at 100 days after HSCT compared to supportive care alone.
• Demographic Consistency: The drug works similarly across different sex, age, and racial groups, although data on non-White participants is limited.
• Market Need: Defitelio addresses a critical need for a treatment option in hepatic VOD, a condition with high mortality rates.
• Regulatory Favorability: The drug was approved under expedited programs, indicating a favorable regulatory environment.
• Safety Profile: Defitelio has a notable safety profile with specific contraindications and potential adverse reactions.

FAQs

What is Defitelio used for?

Defitelio is used to treat hepatic veno-occlusive disease (VOD) with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT)[4].

How effective is Defitelio in clinical trials?

In clinical trials, 38% to 45% of patients treated with Defitelio were alive at 100 days after HSCT, compared to 21% to 31% for patients receiving supportive care alone[1][4].

What are the common adverse reactions of Defitelio?

Common adverse reactions include hypotension, diarrhea, vomiting, nausea, and epistaxis. Major potential adverse reactions include hemorrhage and hypersensitivity reactions[4].

Can Defitelio be used with anticoagulants or fibrinolytic therapies?

No, the use of Defitelio is contraindicated in patients being treated concurrently with anticoagulants or fibrinolytic therapies due to its profibrinolytic activity[4].

What is the recommended dosage of Defitelio?

The recommended dose is 6.25 mg/kg intravenously every 6 hours, given as a 2-hour infusion for at least 21 days, and continued until VOD resolution or up to 60 days of treatment[4].

Sources

1. FDA: Drug Trials Snapshots: DEFITELIO - FDA
2. Jazz Pharmaceuticals: Jazz Pharmaceuticals Announces First Quarter 2024 Financial Results and Affirms 2024 Financial Guidance
3. FDA: 208114Orig1s000 - accessdata.fda.gov
4. FDA: Defitelio (defibrotide sodium) - FDA
5. EMA: Defitelio | European Medicines Agency (EMA)