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The European Commission (EC) has granted marketing authorization for rurioctocog alfa pegol (Adynovi).
Rurioctocog alfa pegol (formerly BAX 855) is a pegylated, full-length, recombinant factor VIII product built on a previously licensed recombinant factor VIII product (Advate).
Rurioctocog alfa pegol is now EC-approved for on-demand and prophylactic use in patients age 12 and older with hemophilia A.
With the EC’s approval, Shire is authorized to market rurioctocog alfa pegol in all member states of the European Union as well as in Iceland, Liechtenstein, and Norway.
The marketing authorization for rurioctocog alfa pegol is based on outcomes from three phase 3 trials—a study of adolescents and adults, a trial of patients undergoing surgical procedures, and a study of pediatric patients.
Trial of adolescents/adults
This study included 137 patients, ages 12 to 58, with previously treated hemophilia A. They were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with rurioctocog alfa pegol. One hundred and twenty-six patients completed the study.
Patients who received prophylaxis had a 90% reduction in annualized bleeding rate (ABR) compared to those who received on-demand treatment. The median ABR was 1.9 in the prophylactic arm and 41.5 in the on-demand arm.
There were 7 adverse events (occurring in 6 patients) that were considered possibly related to rurioctocog alfa pegol. These included diarrhea, nausea, headache, and flushing.
These results were published in Blood in July 2015.
Perioperative study
This study included 15 patients with severe hemophilia A who were undergoing surgical procedures (11 of them major and 4 minor).
The patients received rurioctocog alfa pegol at varying doses and schedules, depending on each patient’s pharmacokinetic profile for major procedures or rurioctocog alfa pegol incremental recovery for minor procedures.
Intra-operative and peri-operative hemostatic efficacy of rurioctocog alfa pegol was deemed “excellent” for all 15 patients. The “excellent” rating meant that blood loss was less than or equal to that expected for the type of procedure performed in a non-hemophilic population.
Post-operatively (day 1 after the procedure), hemostatic efficacy was rated “good” for 1 procedure and “excellent” for the rest. The “good” rating meant that post-operative blood loss was up to 50% more than expected for the type of procedure performed in a non-hemophilic population.
There were no treatment-related adverse events or signs of immunogenicity in this trial.
Results were published in Haemophilia in June 2016.
Pediatric trial
This study included 66 patients, ages 1 to 11, who had previously treated hemophilia A. They received twice-weekly prophylaxis with rurioctocog alfa pegol (50 ± 10 IU/kg) for at least 6 months or 50 exposure days, whichever occurred last.
The median ABR was 2.0, and 38% of patients did not have any bleeding episodes.
None of the patients developed inhibitory antibodies, and there were no treatment-related adverse events.
Results from this trial were presented at the World Federation of Hemophilia 2016 World Congress in July 2016 and published in Haemophilia in November 2016. 
The European Commission (EC) has granted marketing authorization for rurioctocog alfa pegol (Adynovi).
Rurioctocog alfa pegol (formerly BAX 855) is a pegylated, full-length, recombinant factor VIII product built on a previously licensed recombinant factor VIII product (Advate).
Rurioctocog alfa pegol is now EC-approved for on-demand and prophylactic use in patients age 12 and older with hemophilia A.
With the EC’s approval, Shire is authorized to market rurioctocog alfa pegol in all member states of the European Union as well as in Iceland, Liechtenstein, and Norway.
The marketing authorization for rurioctocog alfa pegol is based on outcomes from three phase 3 trials—a study of adolescents and adults, a trial of patients undergoing surgical procedures, and a study of pediatric patients.
Trial of adolescents/adults
This study included 137 patients, ages 12 to 58, with previously treated hemophilia A. They were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with rurioctocog alfa pegol. One hundred and twenty-six patients completed the study.
Patients who received prophylaxis had a 90% reduction in annualized bleeding rate (ABR) compared to those who received on-demand treatment. The median ABR was 1.9 in the prophylactic arm and 41.5 in the on-demand arm.
There were 7 adverse events (occurring in 6 patients) that were considered possibly related to rurioctocog alfa pegol. These included diarrhea, nausea, headache, and flushing.
These results were published in Blood in July 2015.
Perioperative study
This study included 15 patients with severe hemophilia A who were undergoing surgical procedures (11 of them major and 4 minor).
The patients received rurioctocog alfa pegol at varying doses and schedules, depending on each patient’s pharmacokinetic profile for major procedures or rurioctocog alfa pegol incremental recovery for minor procedures.
Intra-operative and peri-operative hemostatic efficacy of rurioctocog alfa pegol was deemed “excellent” for all 15 patients. The “excellent” rating meant that blood loss was less than or equal to that expected for the type of procedure performed in a non-hemophilic population.
Post-operatively (day 1 after the procedure), hemostatic efficacy was rated “good” for 1 procedure and “excellent” for the rest. The “good” rating meant that post-operative blood loss was up to 50% more than expected for the type of procedure performed in a non-hemophilic population.
There were no treatment-related adverse events or signs of immunogenicity in this trial.
Results were published in Haemophilia in June 2016.
Pediatric trial
This study included 66 patients, ages 1 to 11, who had previously treated hemophilia A. They received twice-weekly prophylaxis with rurioctocog alfa pegol (50 ± 10 IU/kg) for at least 6 months or 50 exposure days, whichever occurred last.
The median ABR was 2.0, and 38% of patients did not have any bleeding episodes.
None of the patients developed inhibitory antibodies, and there were no treatment-related adverse events.
Results from this trial were presented at the World Federation of Hemophilia 2016 World Congress in July 2016 and published in Haemophilia in November 2016.
The European Commission (EC) has granted marketing authorization for rurioctocog alfa pegol (Adynovi).
Rurioctocog alfa pegol (formerly BAX 855) is a pegylated, full-length, recombinant factor VIII product built on a previously licensed recombinant factor VIII product (Advate).
Rurioctocog alfa pegol is now EC-approved for on-demand and prophylactic use in patients age 12 and older with hemophilia A.
With the EC’s approval, Shire is authorized to market rurioctocog alfa pegol in all member states of the European Union as well as in Iceland, Liechtenstein, and Norway.
The marketing authorization for rurioctocog alfa pegol is based on outcomes from three phase 3 trials—a study of adolescents and adults, a trial of patients undergoing surgical procedures, and a study of pediatric patients.
Trial of adolescents/adults
This study included 137 patients, ages 12 to 58, with previously treated hemophilia A. They were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with rurioctocog alfa pegol. One hundred and twenty-six patients completed the study.
Patients who received prophylaxis had a 90% reduction in annualized bleeding rate (ABR) compared to those who received on-demand treatment. The median ABR was 1.9 in the prophylactic arm and 41.5 in the on-demand arm.
There were 7 adverse events (occurring in 6 patients) that were considered possibly related to rurioctocog alfa pegol. These included diarrhea, nausea, headache, and flushing.
These results were published in Blood in July 2015.
Perioperative study
This study included 15 patients with severe hemophilia A who were undergoing surgical procedures (11 of them major and 4 minor).
The patients received rurioctocog alfa pegol at varying doses and schedules, depending on each patient’s pharmacokinetic profile for major procedures or rurioctocog alfa pegol incremental recovery for minor procedures.
Intra-operative and peri-operative hemostatic efficacy of rurioctocog alfa pegol was deemed “excellent” for all 15 patients. The “excellent” rating meant that blood loss was less than or equal to that expected for the type of procedure performed in a non-hemophilic population.
Post-operatively (day 1 after the procedure), hemostatic efficacy was rated “good” for 1 procedure and “excellent” for the rest. The “good” rating meant that post-operative blood loss was up to 50% more than expected for the type of procedure performed in a non-hemophilic population.
There were no treatment-related adverse events or signs of immunogenicity in this trial.
Results were published in Haemophilia in June 2016.
Pediatric trial
This study included 66 patients, ages 1 to 11, who had previously treated hemophilia A. They received twice-weekly prophylaxis with rurioctocog alfa pegol (50 ± 10 IU/kg) for at least 6 months or 50 exposure days, whichever occurred last.
The median ABR was 2.0, and 38% of patients did not have any bleeding episodes.
None of the patients developed inhibitory antibodies, and there were no treatment-related adverse events.
Results from this trial were presented at the World Federation of Hemophilia 2016 World Congress in July 2016 and published in Haemophilia in November 2016.