PLAINSBORO, NJ, July 29, 2022 – Novo Nordisk announced today that the US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for REBINYN®, Coagulation Factor IX (Recombinant), GlycoPEGylated, a recombinant DNA-derived coagulation factor IX concentrate, for use in adults and children with hemophilia B (congenital factor IX deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes.1
REBINYN®, an extended half-life treatment used to replace clotting factor IX (FIX), was FDA approved in 2017 for on-demand treatment and control of bleeding episodes, and perioperative management of bleeding in adults and children. The expanded indication will allow for prophylactic treatment resulting in high FIX levels and reduced risk of bleeding. This is important as prophylaxis can help people with Hemophilia participate in physical and social activities with the intent to prevent bleeding.2
“Novo Nordisk continues its long-standing commitment to drive change for the rare blood disorders community. This important milestone for REBINYN® allows patients with Hemophilia B of all ages to maintain high factor levels until their next weekly dose.” said Melissa Leichter, Vice President & Commercial Lead, Rare Disease of Novo Nordisk.
The safety and efficacy of REBINYN® was evaluated in five multi-center, non-controlled, open label trials in on-demand treatment of bleeding episodes, perioperative management of major and minor surgery, and routine prophylaxis or pharmacokinetic evaluation in 115 previously treated patients (PTPs) with Hemophilia B. A total of 15,167 injections were administered over a median of 733 days, equivalent to 15,137 exposure days. This corresponds to 292 patient-years.
Previously treated adults and adolescents (N=29) taking REBINYN® 40 IU/kg once weekly for 1 year in the clinical trial program experienced an overall median annualized bleeding rate (ABR) of 1.04 along with 0 observed inhibitors or thrombotic events. Common adverse reactions (≥1%) in PTPs reported in clinical trials for REBINYN® were itching and injection site reactions.1
“In clinical studies, when adults and adolescent people living with Hemophilia B were given prophylactic treatment with REBINYN®, patients experienced factor IX activity in the same range as people without hemophilia (above 40%) for nearly 80% of the week” said Allison P. Wheeler, MD, MSCI, Vanderbilt University Medical Center, Nashville, TN. “This is supported by the World Federation of Hemophilia acknowledgement that replacement therapy with extended half-life FIX allows for more ambitious prophylaxis.”
About REBINYN®
https://www.rebinyn.com/
REBINYN®, Coagulation Factor IX (Recombinant), GlycoPEGylated, is an extended half-life factor IX molecule for replacement therapy in patients with Hemophilia B. In five multi-center, prospective, non-controlled, open-label clinical trials, 115 PTPs received at least one dose of REBINYN® as part of routine prophylaxis, on-demand treatment of bleeding episodes, perioperative management of major and minor surgery, or pharmacokinetic evaluation. A PTP was defined as a subject with a history of at least 150 exposure days to other Factor IX products (adolescent/adult subjects) or 50 exposure days to other Factor IX products (pediatric subjects), and no history of inhibitors. A total of 15,167 injections were administered over a median of 733 days (range: 29- 2951 days), equivalent to 15,137 exposure days and 292 patient-years.