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defibrotide

Development stage: Approved
Lead developer: Jazz Pharmaceuticals
Modality: miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Recombinant Proteins and Enzymes, Natural RNA Aptamers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Vaccines → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Spiegelmers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, Unmodified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Vaccine mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Long Non-coding RNA (lncRNA) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Protein Replacement mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, miRNA Inhibitors → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapy Plasmids → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravenous

Overview

Defibrotide is a mixture of single-stranded oligodeoxyribonucleotides derived from porcine intestinal mucosal DNA. It is classified as an antithrombotic and profibrinolytic agent, primarily used to treat hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) in both adults and children. Its mechanism involves enhancing fibrinolysis by increasing tissue plasminogen activator and thrombomodulin expression, while decreasing von Willebrand factor and plasminogen activator inhibitor 1 expression. This results in increased breakdown of blood clots without significant systemic anticoagulant effects. Defibrotide also exhibits anti-inflammatory and anti-ischaemic properties[1][2][4][5].

Brand names: DefitelioDasovasNoravidProciclide
Other names: polydeoxyribonucleotide sodium saltdefibrotide sodiumdefibrotide free acid (porcine mucosa)defibrotide free acid (porcine)

Targets

PLAT (Tissue-type plasminogen activator)THBD (Thrombomodulin)PAI-1 (Plasminogen activator inhibitor type 1)BB-031 (Von Willebrand factor)

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