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Nonacog beta pegol (N9-GP, Rebinyn®), a recombinant, GlycoPEGylated coagulation factor IX, has been approved by the US Food and Drug Administration (FDA) for on-demand treatment and control of bleeding episodes in adults and children with hemophilia B.
It is also indicated for the perioperative management of bleeding in these individuals. However, it is not indicated for routine prophylaxis or for immune tolerance induction.
NovoNordisk, the manufacturer of the drug, expects the launch of the coagulation factor in the United States to take place in the first half of 2018.
N9-GP earlier this year had been recommended for marketing authorization by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) as Refixia.® On June 6 it was actually granted marketing authorization, which covers all 28 European Union member states.
The FDA based its approval on the efficacy and safety evaluation of 115 previously treated patients across the four paradigmTM clinical trials. Results from the paradigm 4 trial were published in Thrombosis Research in May 2016.
Of 597 new bleeding episodes in 79 of 105 patients, 551 (93%) were resolved successfully with good or excellent ratings by the patients or study site investigator. Forty (7%) were rated as moderate or poor.
Most (87%) were resolved with 1 injection, 60 (10%) with 2 injections, and 16 (3%) with more than 2 injections.
The median dose to treat a bleeding episode was 42.3 IU/kg.
On-demand treatment had a success rate of 95%, with 120 (84%) of the 143 bleeds treated with one injection.
For perioperative management, N9-GP was rated as excellent or good for 13 surgeries, for a success rate of 100%.
Patients received a preoperative dose of 80 IU/kg. No patient required additional doses on the day of surgery.
During the postoperative period, patients required additional 40 IU/kg doses. The mean total consumption of factor in the pre- and postoperative period was 241 IU/kg (range, 8 – 460 IU/kg.
No unexpected postoperative bleeding occurred.
For full prescribing information, see the package insert. 
Nonacog beta pegol (N9-GP, Rebinyn®), a recombinant, GlycoPEGylated coagulation factor IX, has been approved by the US Food and Drug Administration (FDA) for on-demand treatment and control of bleeding episodes in adults and children with hemophilia B.
It is also indicated for the perioperative management of bleeding in these individuals. However, it is not indicated for routine prophylaxis or for immune tolerance induction.
NovoNordisk, the manufacturer of the drug, expects the launch of the coagulation factor in the United States to take place in the first half of 2018.
N9-GP earlier this year had been recommended for marketing authorization by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) as Refixia.® On June 6 it was actually granted marketing authorization, which covers all 28 European Union member states.
The FDA based its approval on the efficacy and safety evaluation of 115 previously treated patients across the four paradigmTM clinical trials. Results from the paradigm 4 trial were published in Thrombosis Research in May 2016.
Of 597 new bleeding episodes in 79 of 105 patients, 551 (93%) were resolved successfully with good or excellent ratings by the patients or study site investigator. Forty (7%) were rated as moderate or poor.
Most (87%) were resolved with 1 injection, 60 (10%) with 2 injections, and 16 (3%) with more than 2 injections.
The median dose to treat a bleeding episode was 42.3 IU/kg.
On-demand treatment had a success rate of 95%, with 120 (84%) of the 143 bleeds treated with one injection.
For perioperative management, N9-GP was rated as excellent or good for 13 surgeries, for a success rate of 100%.
Patients received a preoperative dose of 80 IU/kg. No patient required additional doses on the day of surgery.
During the postoperative period, patients required additional 40 IU/kg doses. The mean total consumption of factor in the pre- and postoperative period was 241 IU/kg (range, 8 – 460 IU/kg.
No unexpected postoperative bleeding occurred.
For full prescribing information, see the package insert.
Nonacog beta pegol (N9-GP, Rebinyn®), a recombinant, GlycoPEGylated coagulation factor IX, has been approved by the US Food and Drug Administration (FDA) for on-demand treatment and control of bleeding episodes in adults and children with hemophilia B.
It is also indicated for the perioperative management of bleeding in these individuals. However, it is not indicated for routine prophylaxis or for immune tolerance induction.
NovoNordisk, the manufacturer of the drug, expects the launch of the coagulation factor in the United States to take place in the first half of 2018.
N9-GP earlier this year had been recommended for marketing authorization by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) as Refixia.® On June 6 it was actually granted marketing authorization, which covers all 28 European Union member states.
The FDA based its approval on the efficacy and safety evaluation of 115 previously treated patients across the four paradigmTM clinical trials. Results from the paradigm 4 trial were published in Thrombosis Research in May 2016.
Of 597 new bleeding episodes in 79 of 105 patients, 551 (93%) were resolved successfully with good or excellent ratings by the patients or study site investigator. Forty (7%) were rated as moderate or poor.
Most (87%) were resolved with 1 injection, 60 (10%) with 2 injections, and 16 (3%) with more than 2 injections.
The median dose to treat a bleeding episode was 42.3 IU/kg.
On-demand treatment had a success rate of 95%, with 120 (84%) of the 143 bleeds treated with one injection.
For perioperative management, N9-GP was rated as excellent or good for 13 surgeries, for a success rate of 100%.
Patients received a preoperative dose of 80 IU/kg. No patient required additional doses on the day of surgery.
During the postoperative period, patients required additional 40 IU/kg doses. The mean total consumption of factor in the pre- and postoperative period was 241 IU/kg (range, 8 – 460 IU/kg.
No unexpected postoperative bleeding occurred.
For full prescribing information, see the package insert.