Novo Nordisk recently announced that the U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application (sBLA) for REBINYN®, the company’s recombinant, GlycoPEGylated extended half-life therapy. The approval is specific to use in adults and children with hemophilia B (congenital factor IX deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes.

PEGylated products are developed through the binding of polyethylene glycol (PEG) molecules to proteins, which refines the therapy to boost its pharmacokinetic (PK) effect. PKs are an important measurement of the concentration and duration of a drug’s effect in the body. In this case, REBINYN® is designed to prolong the circulating half-life of factor IX to ensure more sustained protection from bleeding and less frequent infusions for hemophilia B patients.  

The new approval represents an expanded indication, as REBINYN® first received FDA approval in May of 2017 for on-demand treatment and control of bleeding episodes, and the perioperative management of bleeding in adults and children with hemophilia B.

According to a new company press release, the safety and efficacy of REBINYN® was evaluated in five multi-center trials 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, which corresponds to 292 patient years. Previously treated adults and adolescents taking the therapy once weekly for one year program experienced an overall median annualized bleeding rate (ABR) of 1.04 along with 0 observed inhibitors or thrombotic events. Common adverse reactions in PTPs reported in clinical trials 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.”

Source: Novo Nordisk press release dated August 3, 2022