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Drug

Defibrotide

Established medicine
ClassAdenosine A1 receptor agonistResearchOngoing clinical research

Also known as DEFIBROTIDE FREE ACID, Defitelio.

RxNorm1311089ChEMBLCHEMBL2108396ATCB01AX01UNII438HCF2X0M
1
Research papers
3
Active clinical trials
5
Protein targets
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

The Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents, and Young Adults With Sickle Cell Disease or Beta Thalassemia Following Myeloablative Conditioning (MAC) and Haploidentical or Matched Unrelated Donor (MUD) Stem Cell Transplantation Utilizing CD34 Enrichment and T-Cell (CD3) Addback

Clinical trial2026-07-27Results expected Q4 2026 · ClinicalTrials.gov

A Single-Arm Safety and Feasibility Study of Defibrotide for the Treatment of Severe COVID-19

Clinical trial2026-07-20Results expected Q4 2026 · ClinicalTrials.gov

A Phase II Intrapatient Open-Label Dose Escalation Trial of Defibrotide in Hematopoietic Cell Transplantation (HCT) Recipients With Sinusoidal Obstructive Syndrome (SOS) Post-HCT Associated With Either Renal and/or Pulmonary Dysfunction With Either Refractory or Progressive Disease Following Defibrotide Therapy

Clinical trial2026-04-15Results expected Q3 2027 · ClinicalTrials.gov

A Phase II Study to Determine the Safety of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

Clinical trial2023-11-07Completed · ClinicalTrials.gov

A Pilot Study to Determine the Safety of Defibrotide in Children With High Risk Kawasaki Disease

Clinical trial2023-10-24Primary completion · ClinicalTrials.gov

Defibrotide Therapy for SARS-CoV2 Acute Respiratory Distress Syndrome (ARDS)

Clinical trial2023-05-09Results posted · ClinicalTrials.gov

Phase IIb Prospective, Multi-center, Randomized, Parallel, Double Blind, Placebo Controlled Trial to Evaluate Defibrotide Intravenous Infusion in the Prevention and Treatment of COVID-19 Respiratory Distress and Cytokine Release Syndrome

Clinical trial2023-02-28Completed · ClinicalTrials.gov

Phase II Open Label Study to Assess Efficacy of 5-day Defibrotide Treatment for Hepatic SOS/VOD

Clinical trial2022-05-01Primary completion · ClinicalTrials.gov

A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant

Clinical trial2022-03-02Results posted · ClinicalTrials.gov

A Pilot Trial of Using Pre-Transplant Risk Stratification and Prophylactic Defibrotide to Prevent Serious Thrombotic Microangiopathy in High-Risk Hematopoietic Stem Cell Transplant Patients

Clinical trial2021-09-16Results posted · ClinicalTrials.gov

Approval: Defitelio (EMA)

Regulatory2013-10-18EMA

Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome.

Research2002-08-01Blood

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Defibrotide
Aliases & brands
DEFIBROTIDE FREE ACIDDefitelio
RxNorm CUI
1311089
ChEMBL ID
CHEMBL2108396
ATC codes
B01AX01
UNII
438HCF2X0M
Primary mechanism
Adenosine A1 receptor agonist
Regulatory jurisdictions
ema

Pharmacology & targets

5 targets

Known molecular targets and mechanisms supported by curated pharmacology databases.

Adenosine receptor A1
ADORA1 · P30542
AGONISTAdenosine A1 receptor agonist
Adenosine receptor A2b
ADORA2B · P29275
AGONISTAdenosine A2 receptor agonist
Adenosine receptor A2a
ADORA2A · P29274
AGONISTAdenosine A2 receptor agonist
Prothrombin
F2 · P00734
ANTAGONISTThrombin antagonist
Plasminogen
PLG · P00747
ACTIVATORPlasminogen activator

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2013-10-18
Latest approval
2013-10-18
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2013-10-18Approval
Approval: Defitelio (EMA)
Indication: Defitelio is indicated for the treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell… Show full indication

Defitelio is indicated for the treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy. It is indicated in adults and in adolescents, children and infants over 1 month of age.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

20 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
20
registered trials across all phases
LATEST COMPLETION 2023
3
Active studies
3
Recruiting
5
Late-stage (III+)
13
Completed
4
Discontinued
PHASE DISTRIBUTIONn = 20
Phase 11Phase 1 / 21Phase 213Phase 2 / 31Phase 33Phase 41

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

Defibrotide in the Human Endotoxemia Model -- an Exploratory Trial Investigating the Effects and the Mechanisms of Defibrotide

CompletedPH IVBernd Jilman=202018

A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant

CompletedPH IIIJazz Pharmaceuticalsn=3722020

Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study

CompletedPH IIIKey trialJazz Pharmaceuticalsn=1,2062016

Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients: A Historically-Controlled, Multi-Center Phase 3 Study to Determine Safety & Efficacy

CompletedPH IIIJazz Pharmaceuticalsn=1342015

Prospective Randomized Study of the Incidence and Outcome of Veno-Occlusive Disease (VOD) With the Prophylactic Use of Defibrotide (DF) in Pediatric Stem Cell Transplantation

CompletedPH II/IIIEuropean Society for Blood and Marrow Transplantationn=3602009

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase II Study to Determine the Safety of Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

CompletedPH IINew York Medical Collegen=202023

Phase IIb Prospective, Multi-center, Randomized, Parallel, Double Blind, Placebo Controlled Trial to Evaluate Defibrotide Intravenous Infusion in the Prevention and Treatment of COVID-19 Respiratory Distress and Cytokine Release Syndrome

CompletedPH IIFundacion para la Formacion e Investigacion Sanitarias de la Region de Murcian=1562023

Use of Defibrotide to Reduce Progression of Acute Respiratory Failure Rate in Patients With COVID-19 Pneumonia

CompletedPH IIIRCCS San Raffaelen=502021

Defibrotide Therapy for SARS-CoV2 Acute Respiratory Distress Syndrome (ARDS)

CompletedPH IGregory Yanikn=132021

A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant

CompletedPH IIIJazz Pharmaceuticalsn=3722020

A Pilot Trial of Using Pre-Transplant Risk Stratification and Prophylactic Defibrotide to Prevent Serious Thrombotic Microangiopathy in High-Risk Hematopoietic Stem Cell Transplant Patients

CompletedPH IIUniversity of California, San Franciscon=252020

Research activity

1 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Journals, researchers & institutions
Top journals
  • Blood1
Leading researchers
  • Antin JH1
  • Arai S1
  • Avigan D1
  • Bearman SI1
  • Chen AR1
  • Elias AD1
  • Guinan EC1
  • Hoppensteadt D1
Leading institutions
free-text, unnormalised
  • Dana-Farber Cancer Institute1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.