User login
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for vonicog alfa (Veyvondi), a recombinant von Willebrand factor (rVWF) product.
The CHMP is recommending vonicog alfa for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD) when desmopressin treatment alone is ineffective or not indicated.
The CHMP’s recommendation will be reviewed by the European Commission, which has the authority to approve medicines for use in the European Union, Norway, Iceland, and Liechtenstein.
The European Commission usually makes a decision within 67 days of the CHMP’s recommendation.
The CHMP’s opinion of vonicog alfa is supported by a pair of phase 3 trials.
Non-surgical setting
Results from a phase 3 trial of vonicog alfa in a non-surgical setting were published in Blood in 2015.
The study included 49 patients with VWD who received vonicog alfa with or without recombinant factor VIII (FVIII).
All participants had successful treatment of bleeding episodes. Most (96.9%) treated bleeds (n=192 bleeds in 22 patients) were given an “excellent” efficacy rating (as good as or better than expected).
Most bleeds (81.8%) were resolved with a single infusion of vonicog alfa, and the treatment had a mean half-life of 21.9 hours.
There were 8 adverse events (AEs) considered related to vonicog alfa, and 2 were serious. One patient experienced 2 simultaneous serious AEs—chest discomfort and increased heart rate—but these were resolved.
There were no thrombotic events in this trial, no treatment-related binding or neutralizing antibodies against VWF, and no neutralizing antibodies against FVIII.
Surgical setting
Results from the phase 3 trial in a surgical setting were presented at the WFH 2018 World Congress.
The trial enrolled 15 adults with severe VWD who were undergoing elective surgical procedures (10 of them major procedures).
Patients received vonicog alfa at 40 to 60 IU per kg of body weight 12 to 24 hours before surgery. Within 3 hours of surgery, each patient’s FVIII level (FVIII:C) was assessed, with a target of 30 IU/dL for minor surgeries and 60 IU/dL for major surgeries.
Within an hour of surgery, patients received a dose of vonicog alfa, with or without recombinant factor VIII, depending on the target FVIII:C levels at the 3-hour assessment.
Ten patients received rVWF alone, 12 did not receive any preoperative FVIII, and 2 did not receive rVWF postoperatively.
The study’s primary endpoint was met. Vonicog alfa demonstrated overall hemostatic efficacy, as assessed 24 hours after the last perioperative infusion or the completion of the study visit, whichever occurred earlier.
Intra- and post-operative hemostasis was rated as “excellent” (as good as or better than expected) in 60% of patients and “good” (probably as good as expected) in 40% of patients.
One patient developed deep vein thrombosis 3 days after undergoing hip replacement surgery.
One patient tested positive for binding antibodies to VWF. None of the patients developed binding antibodies against potential impurities such as rFurin, CHO-protein, or mouse IgG.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for vonicog alfa (Veyvondi), a recombinant von Willebrand factor (rVWF) product.
The CHMP is recommending vonicog alfa for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD) when desmopressin treatment alone is ineffective or not indicated.
The CHMP’s recommendation will be reviewed by the European Commission, which has the authority to approve medicines for use in the European Union, Norway, Iceland, and Liechtenstein.
The European Commission usually makes a decision within 67 days of the CHMP’s recommendation.
The CHMP’s opinion of vonicog alfa is supported by a pair of phase 3 trials.
Non-surgical setting
Results from a phase 3 trial of vonicog alfa in a non-surgical setting were published in Blood in 2015.
The study included 49 patients with VWD who received vonicog alfa with or without recombinant factor VIII (FVIII).
All participants had successful treatment of bleeding episodes. Most (96.9%) treated bleeds (n=192 bleeds in 22 patients) were given an “excellent” efficacy rating (as good as or better than expected).
Most bleeds (81.8%) were resolved with a single infusion of vonicog alfa, and the treatment had a mean half-life of 21.9 hours.
There were 8 adverse events (AEs) considered related to vonicog alfa, and 2 were serious. One patient experienced 2 simultaneous serious AEs—chest discomfort and increased heart rate—but these were resolved.
There were no thrombotic events in this trial, no treatment-related binding or neutralizing antibodies against VWF, and no neutralizing antibodies against FVIII.
Surgical setting
Results from the phase 3 trial in a surgical setting were presented at the WFH 2018 World Congress.
The trial enrolled 15 adults with severe VWD who were undergoing elective surgical procedures (10 of them major procedures).
Patients received vonicog alfa at 40 to 60 IU per kg of body weight 12 to 24 hours before surgery. Within 3 hours of surgery, each patient’s FVIII level (FVIII:C) was assessed, with a target of 30 IU/dL for minor surgeries and 60 IU/dL for major surgeries.
Within an hour of surgery, patients received a dose of vonicog alfa, with or without recombinant factor VIII, depending on the target FVIII:C levels at the 3-hour assessment.
Ten patients received rVWF alone, 12 did not receive any preoperative FVIII, and 2 did not receive rVWF postoperatively.
The study’s primary endpoint was met. Vonicog alfa demonstrated overall hemostatic efficacy, as assessed 24 hours after the last perioperative infusion or the completion of the study visit, whichever occurred earlier.
Intra- and post-operative hemostasis was rated as “excellent” (as good as or better than expected) in 60% of patients and “good” (probably as good as expected) in 40% of patients.
One patient developed deep vein thrombosis 3 days after undergoing hip replacement surgery.
One patient tested positive for binding antibodies to VWF. None of the patients developed binding antibodies against potential impurities such as rFurin, CHO-protein, or mouse IgG.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for vonicog alfa (Veyvondi), a recombinant von Willebrand factor (rVWF) product.
The CHMP is recommending vonicog alfa for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults (age 18 and older) with von Willebrand disease (VWD) when desmopressin treatment alone is ineffective or not indicated.
The CHMP’s recommendation will be reviewed by the European Commission, which has the authority to approve medicines for use in the European Union, Norway, Iceland, and Liechtenstein.
The European Commission usually makes a decision within 67 days of the CHMP’s recommendation.
The CHMP’s opinion of vonicog alfa is supported by a pair of phase 3 trials.
Non-surgical setting
Results from a phase 3 trial of vonicog alfa in a non-surgical setting were published in Blood in 2015.
The study included 49 patients with VWD who received vonicog alfa with or without recombinant factor VIII (FVIII).
All participants had successful treatment of bleeding episodes. Most (96.9%) treated bleeds (n=192 bleeds in 22 patients) were given an “excellent” efficacy rating (as good as or better than expected).
Most bleeds (81.8%) were resolved with a single infusion of vonicog alfa, and the treatment had a mean half-life of 21.9 hours.
There were 8 adverse events (AEs) considered related to vonicog alfa, and 2 were serious. One patient experienced 2 simultaneous serious AEs—chest discomfort and increased heart rate—but these were resolved.
There were no thrombotic events in this trial, no treatment-related binding or neutralizing antibodies against VWF, and no neutralizing antibodies against FVIII.
Surgical setting
Results from the phase 3 trial in a surgical setting were presented at the WFH 2018 World Congress.
The trial enrolled 15 adults with severe VWD who were undergoing elective surgical procedures (10 of them major procedures).
Patients received vonicog alfa at 40 to 60 IU per kg of body weight 12 to 24 hours before surgery. Within 3 hours of surgery, each patient’s FVIII level (FVIII:C) was assessed, with a target of 30 IU/dL for minor surgeries and 60 IU/dL for major surgeries.
Within an hour of surgery, patients received a dose of vonicog alfa, with or without recombinant factor VIII, depending on the target FVIII:C levels at the 3-hour assessment.
Ten patients received rVWF alone, 12 did not receive any preoperative FVIII, and 2 did not receive rVWF postoperatively.
The study’s primary endpoint was met. Vonicog alfa demonstrated overall hemostatic efficacy, as assessed 24 hours after the last perioperative infusion or the completion of the study visit, whichever occurred earlier.
Intra- and post-operative hemostasis was rated as “excellent” (as good as or better than expected) in 60% of patients and “good” (probably as good as expected) in 40% of patients.
One patient developed deep vein thrombosis 3 days after undergoing hip replacement surgery.
One patient tested positive for binding antibodies to VWF. None of the patients developed binding antibodies against potential impurities such as rFurin, CHO-protein, or mouse IgG.