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 15 indications[4].[1]

Defitelio is one of 339 small molecule programs targeting Acute Chest Syndrome. It is currently in Approved.

Indications

Total Trials

Active Trials

338

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

Acute Chest SyndromeActive, not recruiting

NeurotoxicityActive, not recruiting

Diabetic NephropathyApproved

Acute Graft Vs. Host DiseaseApproved

Mucocutaneous Lymph Node SyndromeApproved

PneumoniaActive, not recruiting

Recurrent ThrombophlebitisActive, not recruiting

Thrombotic MicroangiopathyActive, not recruiting

Liver DiseaseActive, not recruiting

Multiple MyelomaActive, not recruiting

Acute Chest SyndromePhase 2

Active, not recruiting

NeurotoxicityPhase 2

Active, not recruiting

Diabetic NephropathyApproved

Approved

Acute Graft Vs. Host DiseaseApproved

Approved

Mucocutaneous Lymph Node SyndromeApproved

Approved

PneumoniaPhase 2

Active, not recruiting

Recurrent ThrombophlebitisApproved

Active, not recruiting

Thrombotic MicroangiopathyPhase 2

Active, not recruiting

Liver DiseasePreclinical

Active, not recruiting

Multiple MyelomaPhase 1/Phase 2

Active, not recruiting

Showing 10 of 15 indications

Competition

Competitive Landscape

1931 competitors in COVID-19

#DrugCompanyPhaseTrials

Defitelio

Jazz Pharmaceuticals

Approved

1 2

Spikevax

Moderna

Preclinical

21 3

Bnt162b2

BioNTech

Preclinical

29 4

Angiomax

BBaxter Healthcare Corporation

Preclinical

2 5

Rayaldee

OOrganon and Co

Preclinical

1 6

Aquasol E

NNational Eye Institute (NEI)

Preclinical

1 7

Statin

Bristol-Myers Squibb

Preclinical

1 8

Statins

Sanofi

Preclinical

1 9

Orap

Teva

Preclinical

1 10

Calcium

Eli Lilly

Preclinical

—

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

Timeline

Expected Milestones

Q3 2027

Phase 2Recruiting

HSCT

NCT05987124

Q4 2027

Phase 2Recruiting

Sickle Cell Disease

NCT02675959

Clinical

Trials by Indication[1]

Hepatic Veno-Occlusive Disease3 trials

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

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

Liver2 trials

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

No longer available·EEmory University··NCT00263978

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

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

Pediatric1 trial

Pediatric Trial Investigating the Incidence & Outcome of Veno-Occlusive Disease With the Prophylactic Use of Defibrotide

Completed·EEuropean Society for Blood and Marrow Transplantation·Phase 2/3·NCT00272948·360 enrolled

Sickle Cell Disease1 trial

Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

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

HSCT1 trial

Defibrotide Dose-escalation for SOS Post-HSCT

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

14 more indications

COVID-191 trial

Defibrotide for the Treatment of Severe COVID-19

Recruiting·—·Phase 2·NCT04652115·42 enrolled

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

Prophylaxis1 trial

Defibrotide TMA Prophylaxis Pilot Trial

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

Acute Chest Syndrome1 trial

Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

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

Kawasaki Disease1 trial

Defibrotide in Children With High Risk Kawasaki Disease

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

Cytokine Release Syndrome1 trial

Defibrotide as Prevention and Treatment of Respiratory Distress and Cytokine Release Syndrome of Covid 19.

Completed·FFundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia·Phase 2·NCT04348383·156 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

Multiple Myeloma1 trial

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

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

Thrombotic Microangiopathy1 trial

Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy for Neuroblastoma

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

Pneumonia1 trial

Defibrotide in COVID-19 Pneumonia

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

Treatment1 trial

5-day Defibrotide Treatment for Hepatic SOS/VOD

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

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

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

Q3 2027

Q4 2027

Trials by Indication[1]

Hepatic Veno-Occlusive Disease3 trials

Liver2 trials

Pediatric1 trial

Sickle Cell Disease1 trial

HSCT1 trial

COVID-191 trial

Endotoxemia1 trial

Prophylaxis1 trial

Acute Chest Syndrome1 trial

Kawasaki Disease1 trial

Cytokine Release Syndrome1 trial

Prevention1 trial

Multiple Myeloma1 trial

Thrombotic Microangiopathy1 trial

Pneumonia1 trial

Treatment1 trial

Acute Respiratory Distress Syndrome1 trial

Neurotoxicity1 trial

Stem Cell Transplantation1 trial

FDA Approval Timeline[5]

References

Drug Info

Quick Facts

Patent Landscape[6]

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