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Defitelio FDA Approval History

FDA Approved: Yes (First approved March 30, 2016)
Brand name: Defitelio
Generic name: defibrotide
Dosage form: Injection
Company: Jazz Pharmaceuticals plc
Treatment for: Hepatic Veno-Occlusive Disease

Defitelio (defibrotide sodium) is a deoxyribonucleic acid derivative anticoagulant for the treatment of patients with hepatic veno-occlusive disease (VOD) with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).

The FDA approval of Defitelio was supported by efficacy data from three studies that investigated 528 patients with hepatic VOD with renal or pulmonary dysfunction following HSCT. In the three studies, 38 to 45 percent of patients treated with Defitelio were alive 100 days after HSCT. An analysis of data from published reports indicates Day +100 survival rates for patients receiving only supportive care or interventions other than Defitelio is 21 to 31 percent.

Defitelio is administered via intravenous infusion every six hours for a minimum of 21 days, or up to a maximum of 60 days if the signs and symptoms of hepatic VOD have not resolved. Potentially serious side effects include bleeding and allergic reactions. The most common side effects reported during clinical trials were hypotension, diarrhea, vomiting, nausea and nose bleeds.

Development timeline for Defitelio

Mar 30, 2016Approval FDA Approves Defitelio (defibrotide sodium) for Hepatic Veno-Occlusive Disease Following Hematopoietic Stem-Cell Transplantation
Sep 30, 2015Jazz Pharmaceuticals Announces FDA Acceptance for Filing with Priority Review of NDA for Defibrotide
Dec 11, 2014Jazz Pharmaceuticals Initiates Rolling NDA Submission For Defibrotide For The Treatment Of Severe Hepatic Veno-Occlusive Disease

Further information

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