The Food and Drug Administration (FDA) has expanded the approval of Rebinyn® (coagulation factor IX [recombinant], GlycoPEGylated) to include use in adults and children with hemophilia B for routine prophylaxis to reduce the frequency of bleeding episodes. The recombinant DNA-derived coagulation Factor IX concentrate is also approved for on-demand treatment and control of bleeding episodes and for perioperative management of bleeding.
The approval for routine prophylaxis was supported by data from the main phase of the adult/adolescent and pediatric previously treated patients (PTP) trials. A PTP was defined as a patient with a history of at least 150 exposure days to other Factor IX products (adolescent/adult patients) or 50 exposure days to other Factor IX products (pediatric patients), and no history of inhibitors. Patients 0 to 65 years of age received Rebinyn 40 IU/kg once weekly for approximately 52 weeks.
Results showed an overall median annualized bleeding rate (ABR) of 1.04, 2.00, and 0 in patients 13 years of age and older, 7 to 12 years of age, and 6 years and younger, respectively. The most common adverse reactions reported with Rebinyn were itching and injection site reactions.
“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.”
Rebinyn is available as a lyophilized powder in single-dose vials containing 500 IU, 1000 IU, and 2000 IU.
- Novo Nordisk receives FDA approval for Rebinyn® for routine prophylaxis to prevent bleeding in people living with hemophilia B. News release. Novo Nordisk. July 29, 2022. Accessed August 2, 2022. https://www.novonordisk-us.com/content/nncorp/us/en_us/media/news-archive/news-details.html?id=127305
- Rebinyn. Package insert. Novo Nordisk; 2022. Accessed August 2, 2022. https://www.novo-pi.com/rebinyn.pdf