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Defibrotide Injection
Phosphodiesterase inhibitor
Defibrotide Injection, developed by IRCCS San Raffaele, is a promising treatment for various conditions including thrombotic microangiopathy and severe COVID-19. Currently in multiple Phase 2 trials, it shows potential in preventing and treating respiratory distress and cytokine release syndrome. The drug has no FDA label but is actively being studied for its safety and efficacy.
At a glance
| Generic name | Defibrotide Injection |
|---|---|
| Sponsor | IRCCS San Raffaele |
| Drug class | Phosphodiesterase inhibitor |
| Target | Phosphodiesterase |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 2 |
Mechanism of action
Defibrotide is a mixture of single-stranded oligonucleotides that inhibit the activity of phosphodiesterase, leading to an increase in cyclic AMP levels and a subsequent decrease in platelet activation.
Approved indications
- Hepatic Veno-Occlusive Disease
Common side effects
- Nausea
- Vomiting
- Diarrhea