The FDA has approved the biologics license application for Rebinyn (coagulation factor IX [recombinant)], glycoPEGylated, Novo Nordisk) for on-demand treatment and control of bleeding episodes and the perioperative management of bleeding around the time of surgery in adults and children with hemophilia B. The approval follows a meeting of the agency’s Blood Products Advisory Committee in April.
Rebinyn is an extended half-life factor IX molecule. Novo Nordisk expects to launch the product in the United States in the first half of 2018.
The FDA’s approval of Rebinyn was based on data from a phase 3 clinical program that enrolled children and adults with severe or moderately severe hemophilia B. In this program, 115 previously treated patients had a total of more than 8,800 exposure days for up to 2.7 years of treatment with Rebinyn.
The efficacy evaluation included 105 subjects: 62 adults (18 to 65 years of age); 18 adolescents (13 to 17 years of age); and 25 children (1 to 12 years of age).
A total of 597 bleeding episodes were reported in 79 of the 105 subjects in the clinical program in previously treated patients. Bleeding episodes were treated with Rebinyn 40 IU/kg for minor or moderate bleeds or 80 IU/kg for major bleeds, with additional doses of 40 IU/kg as needed. The median dose to treat a bleeding episode was 42.3 IU/kg.
The efficacy of Rebinyn was rated as “excellent or good” in 93% of the 597 bleeding episodes, and as “moderate or poor” in 7%. Most bleeding episodes (87%) required one injection for treatment, whereas 10% required two injections and 3% required more than two injections.
In a surgery trial, the efficacy analysis of Rebinyn in perioperative management included 13 surgical procedures, of which nine were major and performed in 13 previously treated adolescent and adult patients. The procedures included nine orthopedic, one gastrointestinal, and three in the oral cavity. The hemostatic effect of Rebinyn during surgery was evaluated on a four-point scale of excellent, good, moderate, or poor.
The intraoperative hemostatic effect of Rebinyn was rated as “excellent or good” for the 13 surgeries, for a success rate of 100%. A preoperative dose of 80 IU/kg of Rebinyn was effective, and no subjects required additional doses on the day of surgery. The median number of additional 40-IU/kg doses during the postoperative period was 2.0 for days 1 to 6; 1.5 for days 7 to 13; and 3.0 for days 1 to 13. The mean total consumption of Rebinyn during the pre- and postoperative periods was 241 IU/kg (range: 81 IU/kg to 460 IU/kg). There was no unexpected postoperative bleeding.