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FDA approves Defitelio to treat hepatic VOD, multiorgan dysfunction after HSCT
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The FDA today approved defibrotide sodium to treat adults and children who developed hepatic veno-occlusive disease with renal and pulmonary dysfunction after hematopoietic stem cell transplantation.
The FDA based its decision in part on results from two prospective clinical trials and an expanded access study that evaluated the efficacy of defibrotide sodium (Defitelio, Jazz Pharmaceuticals) in 528 patients with hepatic veno-occlusive disease (VOD) with multiorgan dysfunction after hematopoietic stem cell transplantation (HSCT). Participants received 6.25 mg/kg of defibrotide sodium intravenously every 6 hours until resolution of VOD.
Survival rates after 100 days were 38% (95% CI, 29%-48%) in the first study, 44% (95% CI, 33%-55%) in the second study and 45% (95% CI, 40%-51%) in the third study.
Patients who received supportive care or interventions other than defibrotide sodium demonstrated 100-day survival rates ranging from 21% to 31%.
“The approval of Defitelio fills a significant need in the transplantation community to treat this rare but frequently fatal complication in patients who receive chemotherapy and HSCT,” Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release
The most common adverse events associated with defibrotide sodium include hypotension, diarrhea, vomiting, nausea and epistaxis. Major potential adverse reactions include hemorrhage and hypersensitivity.
The FDA recommends a 6.25-mg/kg dose of defibrotide sodium intravenously every 6 hours as a 2-hour infusion for at least 21 days, continued until VOD resolution or up to 60 days of treatment.