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Photo by Chad McNeeley
The US Food and Drug Administration (FDA) has approved the use of defibrotide sodium (Defitelio).
The product can now be used to treat adult and pediatric patients who develop hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome, with renal or pulmonary dysfunction after receiving a hematopoietic stem cell transplant (HSCT).
Defibrotide sodium is the first FDA-approved therapy for patients with this rare, potentially fatal complication.
Defibrotide sodium is a product of Jazz Pharmaceuticals, Inc. The company said shipments of the drug to distribution channels will begin within a week.
The recommended dose and schedule for defibrotide sodium is 6.25 mg/kg every 6 hours, given as a 2-hour intravenous infusion, for at least 21 days, and continued until VOD resolution or up to 60 days of treatment.
In vitro defibrotide sodium has profibrinolytic activity. The use of defibrotide sodium is contraindicated in patients receiving concurrent anticoagulants or fibrinolytic therapies. Hemorrhage and hypersensitivity reactions are the major potential adverse reactions.
The FDA previously granted the defibrotide sodium application priority review status, and the drug received orphan drug designation from the FDA for the treatment of hepatic VOD.
Full prescribing information for defibrotide sodium can be found on the FDA website.
Trial results
The FDA’s approval of defibrotide sodium is supported by data in 528 patients treated on 3 studies: a phase 2 trial, a phase 3 trial, and an expanded access study. Data from the expanded access study were presented at the 2015 BMT Tandem Meetings, and data from the phase 3 trial were published in Blood earlier this year.
The 528 patients all had hepatic VOD with multi-organ dysfunction after HSCT. They received defibrotide sodium at 6.25 mg/kg intravenously every 6 hours until resolution of VOD.
The approval was based on survival at day +100 after HSCT. The day +100 survival rates for Study 1 (phase 3, n=102), Study 2 (phase 2, n=75), and Study 3 (expanded access, n=351) were 38%, 44%, and 45%, respectively.
Based on published reports and analyses of patient-level data, the day +100 survival rates were 21% to 31% for patients with hepatic VOD with renal or pulmonary dysfunction who received supportive care or interventions other than defibrotide sodium.
The safety of defibrotide sodium to support approval is based on data from 176 patients in the clinical development program for the treatment of VOD with renal and/or pulmonary dysfunction following HSCT.
The most common adverse events (incidence ≥10% and independent of causality) were hypotension, diarrhea, vomiting, nausea, and epistaxis. The most common serious adverse events (incidence ≥5% and independent of causality) were hypotension (11%) and pulmonary alveolar hemorrhage (7%). 
Photo by Chad McNeeley
The US Food and Drug Administration (FDA) has approved the use of defibrotide sodium (Defitelio).
The product can now be used to treat adult and pediatric patients who develop hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome, with renal or pulmonary dysfunction after receiving a hematopoietic stem cell transplant (HSCT).
Defibrotide sodium is the first FDA-approved therapy for patients with this rare, potentially fatal complication.
Defibrotide sodium is a product of Jazz Pharmaceuticals, Inc. The company said shipments of the drug to distribution channels will begin within a week.
The recommended dose and schedule for defibrotide sodium is 6.25 mg/kg every 6 hours, given as a 2-hour intravenous infusion, for at least 21 days, and continued until VOD resolution or up to 60 days of treatment.
In vitro defibrotide sodium has profibrinolytic activity. The use of defibrotide sodium is contraindicated in patients receiving concurrent anticoagulants or fibrinolytic therapies. Hemorrhage and hypersensitivity reactions are the major potential adverse reactions.
The FDA previously granted the defibrotide sodium application priority review status, and the drug received orphan drug designation from the FDA for the treatment of hepatic VOD.
Full prescribing information for defibrotide sodium can be found on the FDA website.
Trial results
The FDA’s approval of defibrotide sodium is supported by data in 528 patients treated on 3 studies: a phase 2 trial, a phase 3 trial, and an expanded access study. Data from the expanded access study were presented at the 2015 BMT Tandem Meetings, and data from the phase 3 trial were published in Blood earlier this year.
The 528 patients all had hepatic VOD with multi-organ dysfunction after HSCT. They received defibrotide sodium at 6.25 mg/kg intravenously every 6 hours until resolution of VOD.
The approval was based on survival at day +100 after HSCT. The day +100 survival rates for Study 1 (phase 3, n=102), Study 2 (phase 2, n=75), and Study 3 (expanded access, n=351) were 38%, 44%, and 45%, respectively.
Based on published reports and analyses of patient-level data, the day +100 survival rates were 21% to 31% for patients with hepatic VOD with renal or pulmonary dysfunction who received supportive care or interventions other than defibrotide sodium.
The safety of defibrotide sodium to support approval is based on data from 176 patients in the clinical development program for the treatment of VOD with renal and/or pulmonary dysfunction following HSCT.
The most common adverse events (incidence ≥10% and independent of causality) were hypotension, diarrhea, vomiting, nausea, and epistaxis. The most common serious adverse events (incidence ≥5% and independent of causality) were hypotension (11%) and pulmonary alveolar hemorrhage (7%).
Photo by Chad McNeeley
The US Food and Drug Administration (FDA) has approved the use of defibrotide sodium (Defitelio).
The product can now be used to treat adult and pediatric patients who develop hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome, with renal or pulmonary dysfunction after receiving a hematopoietic stem cell transplant (HSCT).
Defibrotide sodium is the first FDA-approved therapy for patients with this rare, potentially fatal complication.
Defibrotide sodium is a product of Jazz Pharmaceuticals, Inc. The company said shipments of the drug to distribution channels will begin within a week.
The recommended dose and schedule for defibrotide sodium is 6.25 mg/kg every 6 hours, given as a 2-hour intravenous infusion, for at least 21 days, and continued until VOD resolution or up to 60 days of treatment.
In vitro defibrotide sodium has profibrinolytic activity. The use of defibrotide sodium is contraindicated in patients receiving concurrent anticoagulants or fibrinolytic therapies. Hemorrhage and hypersensitivity reactions are the major potential adverse reactions.
The FDA previously granted the defibrotide sodium application priority review status, and the drug received orphan drug designation from the FDA for the treatment of hepatic VOD.
Full prescribing information for defibrotide sodium can be found on the FDA website.
Trial results
The FDA’s approval of defibrotide sodium is supported by data in 528 patients treated on 3 studies: a phase 2 trial, a phase 3 trial, and an expanded access study. Data from the expanded access study were presented at the 2015 BMT Tandem Meetings, and data from the phase 3 trial were published in Blood earlier this year.
The 528 patients all had hepatic VOD with multi-organ dysfunction after HSCT. They received defibrotide sodium at 6.25 mg/kg intravenously every 6 hours until resolution of VOD.
The approval was based on survival at day +100 after HSCT. The day +100 survival rates for Study 1 (phase 3, n=102), Study 2 (phase 2, n=75), and Study 3 (expanded access, n=351) were 38%, 44%, and 45%, respectively.
Based on published reports and analyses of patient-level data, the day +100 survival rates were 21% to 31% for patients with hepatic VOD with renal or pulmonary dysfunction who received supportive care or interventions other than defibrotide sodium.
The safety of defibrotide sodium to support approval is based on data from 176 patients in the clinical development program for the treatment of VOD with renal and/or pulmonary dysfunction following HSCT.
The most common adverse events (incidence ≥10% and independent of causality) were hypotension, diarrhea, vomiting, nausea, and epistaxis. The most common serious adverse events (incidence ≥5% and independent of causality) were hypotension (11%) and pulmonary alveolar hemorrhage (7%).