Drugs / Defibrotide
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hepatic veno-occlusive disease 2 milestones | |||||
| Filed | US (FDA) | — | hepatic veno-occlusive disease | 2015-07-31 | “completed the rolling submission of the NDA for defibrotide to the U.S. Food and Drug Administration ( FDA ) on July 31, 2015” jazzpharma.com ↗ “FDA has accepted for filing with Priority Review its recently submitted New Drug Application (NDA) for defibrotide.” jazzpharma.com ↗ |
| Filed | US (FDA) | — | hepatic veno-occlusive disease | 2014-12-11 | “initiation of a rolling submission of a New Drug Application (NDA) to the United States (U.S.) Food and Drug Administration ( FDA ) for defibrotide” jazzpharma.com ↗ “acceptance for filing by the U.S. Food and Drug Administration ( FDA ) of the first new drug application (NDA) for defibrotide” jazzpharma.com ↗ “acceptance for filing by the U.S. Food and Drug Administration ( FDA ) of the NDA for defibrotide.” jazzpharma.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | US (FDA) | DEFITELIO | — | 2016-03-30 | fda.gov ↗ |
| Approved | EU (EMA) | Defitelio | — | 2013-10-18 | europa.eu ↗ |
Trials 21
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT04530604 | Oct 2020 → Mar 2021 | COVID-19, acute respiratory distress syndrome | Gregory Yanik | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT00406978 | Feb 2006 → Sep 2010 | plasma cell myeloma | Silvio Aime | Completed | No outcome recorded |
| Phase 213 trials · 2 missed | ||||||
| Phase 2 | NCT06182410 | Aug 2024 → May 2027 | neuroblastoma, thrombotic microangiopathy | University of California, San Francisco | Withdrawn | No outcome recorded Stop: Business |
| Phase 2 | NCT05987124 | Mar 2024 → Aug 2027 expected | hepatic veno-occlusive disease | New York Medical College | Recruiting | No outcome recorded |
| Phase 2 | NCT04313036 | Mar 2021 → May 2022 | disease related to hematopoietic stem cell transplant, hepatic veno-occlusive disease | Loyola University | Unknown | No outcome recorded |
| Phase 2 | NCT04777422 | Feb 2021 → Oct 2023 | Kawasaki disease | New York Medical College | Unknown | No outcome recorded |
| Phase 2 | NCT04652115 | Jan 2021 → Dec 2026 expected | COVID-19 | Brigham and Women's Hospital | Recruiting | No outcome recorded |
| Phase 2 | NCT04335201 | May 2020 → Apr 2021 | — | IRCCS San Raffaele | Completed | No outcome recorded |
| Phase 2 | NCT04348383 | Apr 2020 → Feb 2023 | COVID-19 | Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia | Completed | Missed primary |
| Phase 2 | NCT03954106 | Oct 2019 → Sep 2020 | diffuse large B-cell lymphoma, toxic encephalopathy | Jazz Pharmaceuticals | Terminated | Missed primary |
| Phase 2 | NCT03384693 | May 2018 → Jul 2020 | thrombotic microangiopathy | University of California, San Francisco | Completed | No outcome recorded |
| Phase 2 | NCT03339297 | Feb 2018 → May 2020 | acute graft versus host disease | Jazz Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03805581 | Jan 2018 → Jul 2022 | acute chest syndrome, sickle cell disease | New York Medical College | Completed | No outcome recorded |
| Phase 2 | NCT02675959 | Jul 2017 → Dec 2026 expected | sickle cell disease | New York Medical College | Recruiting | No outcome recorded |
| Phase 2 | NCT00003966 | Apr 2000 → Feb 2006 | hepatic veno-occlusive disease | Dana-Farber Cancer Institute | Completed | No outcome recorded |
| Phase 33 trials · 1 met primary · 1 missed | ||||||
| Phase 3 | NCT02851407 | Sep 2016 → Oct 2020 | hepatic veno-occlusive disease | Jazz Pharmaceuticals | Completed | Missed primary |
| Phase 3 | NCT00628498 | Dec 2007 → Jul 2016 | hepatic veno-occlusive disease | Jazz Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT00358501 | Jul 2006 → Sep 2008 | hepatic veno-occlusive disease | Jazz Pharmaceuticals | Completed | Met primary |
| Phase 41 trial · 1 missed | ||||||
| Phase 4 | NCT02876601 | Apr 2017 → Feb 2018 | — | Medical University of Vienna | Completed | Missed primary |
| Phase not stated2 trials | ||||||
| — | NCT00263978 | Nov 2005 → Jun 2009 | liver disorder | Emory University | No longer available | No outcome recorded |
| — | NCT00143546 | Nov 2003 → ? | hepatic veno-occlusive disease | St. Jude Children's Research Hospital | No longer available | No outcome recorded |
| Comparator or background therapy2 trials | ||||||
| Phase 2/3 | NCT00272948 Comparator | Dec 2005 → Jan 2009 | hepatic veno-occlusive disease | European Society for Blood and Marrow Transplantation | Completed | No outcome recorded |
| Phase 4 | NCT01718288 Background | Nov 2006 → Mar 2011 | peripheral arterial disease | Fadoi Foundation, Italy | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2020-04-29 | Jazz Pharmaceuticals | Results Jazz Pharmaceuticals Stops Enrollment in Phase 3 Study Evaluating Defibrotide for the Prevention of Veno-Occlusive Disease jazzpharma.com ↗
The IDMC recommendation was made after completion of a pre-planned interim analysis of the first 280 patients and where the study met the protocol-specified futility criteria. |
| 2016-08-02 | Jazz Pharmaceuticals | Regulatory CMS Grants New Technology Add-On Payment to Defitelio for the Treatment of Hepatic Veno-Occlusive Disease with Renal or Pulmonary Dysfunction Following Hematopoietic Stem-Cell Transplantation jazzpharma.com ↗
DUBLIN , Aug. 2, 2016 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the United States (U.S.) Centers for Medicare and Medicaid Services (CMS) has granted a New Technology Add-on Payment (NTAP) for Defitelio® (defibrotide sodium), which is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT). |
| 2016-03-30 | Jazz Pharmaceuticals | Regulatory Jazz Pharmaceuticals Announces FDA Approval of Defitelio® (defibrotide sodium) for the Treatment of Hepatic Veno-Occlusive Disease (VOD) with Renal or Pulmonary Dysfunction Following Hematopoietic Stem-Cell Transplantation (HSCT) jazzpharma.com ↗
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the United States (U.S.) Food and Drug Administration ( FDA ) granted marketing approval for Defitelio ® (defibrotide sodium) for the treatment of adult and pediatric patients with hepatic VOD, also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following HSCT. |
| 2016-02-01 | Jazz Pharmaceuticals | Results Results from Phase 3 Trial of Defibrotide for the Treatment of Severe Veno-Occlusive Disease and Multi-Organ Failure Published Online in BLOOD jazzpharma.com ↗ |
| 2015-09-30 | Jazz Pharmaceuticals | Regulatory Jazz Pharmaceuticals Announces U.S. FDA Acceptance for Filing with Priority Review of NDA for Defibrotide for Hepatic Veno-Occlusive Disease jazzpharma.com ↗
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the United States (U.S.) Food and Drug Administration ( FDA ) has accepted for filing with Priority Review its recently submitted New Drug Application (NDA) for defibrotide. |
| 2014-12-11 | Jazz Pharmaceuticals | Regulatory Jazz Pharmaceuticals Initiates Rolling NDA Submission For Defibrotide For The Treatment Of Severe Hepatic Veno-Occlusive Disease jazzpharma.com ↗ |
All press releases naming this drug 17 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 23 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Defibrotide | “standard heparin / low molecular weight heparin, hemorheological / vasodilators such as pentoxifylline / buflomedil, propionyl-L-carnitine, Defibrotide) but not prostanoids” NCT01718288 ↗22“Defibrotide (DF) is a novel orally bioavailable polydisperse oligonucleotide with anti-thrombotic and anti-adhesive effects” NCT00406978 ↗ |
| Known as | Defitelio | “Protection of endothelial dysfunction by intravenous infusion of Defibrotide (Defitelio), expected to decrease inflammation and expression of adhesion molecules in the endothelium” NCT04348383 ↗ |
| Action | Inhibit | “It is a polydeoxyribonucleotide salt that blocks plasminogen activator inhibitor-1 (PAI-1)” NCT03384693 ↗ |
| Modality | Oligonucleotide (other) | “Defibrotide (DF) is a novel orally bioavailable polydisperse oligonucleotide” NCT00406978 ↗ |
| Route | Intravenous | “defibrotide IV over 2 hours every 6 hours” NCT00003966 ↗ |