Defitelio

(Defibrotide)

ApprovedPeptide

Defibrotide is used to treat a serious condition called hepatic sinusoidal obstruction syndrome (also called veno-occlusive disease) that can develop after a bone marrow or stem cell transplant.[2] Defitelio is currently approved and in use across 9 indications[4].[1]

Defitelio is one of 249 small molecule programs targeting Liver Disease. It is currently in Approved.

Indications

Total Trials

Active Trials

248

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

Liver DiseaseActive, not recruiting

Recurrent ThrombophlebitisActive, not recruiting

Acute Graft Vs. Host DiseaseApproved

Diabetic NephropathyApproved

Graft Versus Host DiseaseApproved

Mucocutaneous Lymph Node SyndromeApproved

HSCTRecruiting

Sickle Cell DiseaseRecruiting

COVID-19Recruiting

Liver DiseasePreclinical

Active, not recruiting

Recurrent ThrombophlebitisApproved

Active, not recruiting

Acute Graft Vs. Host DiseaseApproved

Approved

Diabetic NephropathyApproved

Approved

Graft Versus Host DiseaseApproved

Approved

Mucocutaneous Lymph Node SyndromeApproved

Approved

HSCTPhase 2

Recruiting

Sickle Cell DiseasePhase 2

Recruiting

COVID-19Phase 2

Recruiting

Competition

Competitive Landscape

317 competitors in COVID-19

#DrugCompanyPhaseTrials

Defitelio

Jazz Pharmaceuticals

Approved

1 2

Nexavar

Bayer

Preclinical

1 3

Aldactone

Pfizer

Preclinical

1 4

Stivarga

Bayer

Preclinical

1 5

Aimovig

Amgen

Preclinical

1 6

Calciferol

Activella

Angiomax

BBaxter Healthcare Corporation

Preclinical

2 9

Benadryl

PParkedale

Preclinical

1 10

Tricor

JJagotec

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2027

Phase 2Recruiting

COVID-19

NCT04652115

Q3 2027

Phase 2Recruiting

HSCT

NCT05987124

Q4 2027

Phase 2Recruiting

Sickle Cell Disease

NCT02675959

Clinical

Trials by Indication[1]

Hepatic Veno-Occlusive Disease3 trials

Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 3·NCT02851407·372 enrolled

Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 3·NCT00358501·134 enrolled

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

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 3·NCT00628498·1,206 enrolled

Liver2 trials

Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the Liver

No longer available·SSt. Jude Children's Research Hospital··NCT00143546

Defibrotide Compassionate Use for Patients With Life Threatening Veno-Occlusive Disease of the Liver

No longer available·EEmory University··NCT00263978

Endotoxemia1 trial

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

Completed·BBernd Jilma·Phase 4·NCT02876601·20 enrolled

Pneumonia1 trial

Defibrotide in COVID-19 Pneumonia

Completed·IIRCCS San Raffaele·Phase 2·NCT04335201·50 enrolled

Acute Respiratory Distress Syndrome1 trial

Defibrotide Therapy for SARS-CoV2 (COVID-19) Acute Respiratory Distress Syndrome (ARDS)

Completed·GGregory Yanik·Phase 1·NCT04530604·13 enrolled

14 more indications

Neurotoxicity1 trial

A Safety and Efficacy Study of Defibrotide in the Prevention of Chimeric Antigen Receptor-T-cell-associated Neurotoxicity

Terminated· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 2·NCT03954106·25 enrolled

Stem Cell Transplantation1 trial

Defibrotide in Treating Patients With Liver Damage Following Peripheral Stem Cell Transplantation

Completed·—·Phase 2·NCT00003966·151 enrolled

Prevention1 trial

An Open-Label Study of Defibrotide for the Prevention of Acute Graft-versus-Host-Disease (AGvHD)

Completed· Jazz Pharmaceuticals logo

Jazz Pharmaceuticals·Phase 2·NCT03339297·152 enrolled

Kawasaki Disease1 trial

Defibrotide in Children With High Risk Kawasaki Disease

Unknown status·NNew York Medical College·Phase 2·NCT04777422·2 enrolled

Thrombotic Microangiopathy1 trial

Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy for Neuroblastoma

Withdrawn·UUniversity of California, San Francisco·Phase 2·NCT06182410

Prophylaxis1 trial

Defibrotide TMA Prophylaxis Pilot Trial

Completed·UUniversity of California, San Francisco·Phase 2·NCT03384693·25 enrolled

Treatment1 trial

5-day Defibrotide Treatment for Hepatic SOS/VOD

Unknown status·LLoyola University·Phase 2·NCT04313036·24 enrolled

HSCT1 trial

Defibrotide Dose-escalation for SOS Post-HSCT

Recruiting·NNew York Medical College·Phase 2·NCT05987124·20 enrolled

Multiple Myeloma1 trial

Melphalan, Prednisone, Thalidomide and Defibrotide in Relapsed Multiple Myeloma Patients

Completed·SSilvio Aime·Phase 1/2·NCT00406978·24 enrolled

Completed·NNew York Medical College·Phase 2·NCT03805581·20 enrolled

Sickle Cell Disease1 trial

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

Recruiting·NNew York Medical College·Phase 2·NCT02675959·40 enrolled

COVID-191 trial

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

Recruiting·BBrigham and Women's Hospital·Phase 2·NCT04652115·42 enrolled

Regulatory

FDA Approval Timeline[5]

Mar 30, 2016

DEFITELIO

NDA NDA208114

Sources

References

[1]
ClinicalTrials.gov — 22 trial records for Defibrotide. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Defitelio drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Defibrotide compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Defibrotide. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA208114). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Defitelio. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Jazz Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Adenosine A2 Receptor Agonist; Thrombin Antagonist; Plasminogen Activator; Adenosine A1 Receptor Agonist[3]
Modality: Peptide[3]
Highest Phase: Approved[1]
Company: Jazz Pharmaceuticals

Patent Landscape[6]

Patent Cliff

Jun 22, 2032

Earliest patent expiration — generic entry possible after this date

Patent #Expires

11236328Jun 22, 2032

11085043Jun 22, 2032

12529052Jun 22, 2032

11746348Jun 22, 2032

12534722Jun 22, 2032

Defitelio

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q1 2027

Q3 2027

Q4 2027

Trials by Indication[1]

Hepatic Veno-Occlusive Disease3 trials

Liver2 trials

Endotoxemia1 trial

Pneumonia1 trial

Acute Respiratory Distress Syndrome1 trial

Neurotoxicity1 trial

Stem Cell Transplantation1 trial

Prevention1 trial

Kawasaki Disease1 trial

Thrombotic Microangiopathy1 trial

Prophylaxis1 trial

Treatment1 trial

HSCT1 trial

Multiple Myeloma1 trial

Pediatric1 trial

Cytokine Release Syndrome1 trial

Acute Chest Syndrome1 trial

Sickle Cell Disease1 trial

COVID-191 trial

FDA Approval Timeline[5]

References

Drug Info

Quick Facts

Patent Landscape[6]

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