Novo Nordisk recently announced the availability of REBINYN®, the company’s recombinant GlycoPEGylated product for the treatment of adults and children with hemophilia B (factor IX deficiency). The therapy was first approved by the U.S. Food and Drug Administration in May of 2017.
REBINYN® is indicated for on-demand treatment and control of bleeding episodes, and the perioperative management of bleeding in adults and children with hemophilia B. It is not indicated for routine prophylaxis or for immune tolerance induction in patients with hemophilia B.
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.
REBINYN® was evaluated in the paradigm™ clinical trial program. In the on-demand arm of the adolescent and adult clinical trial, the therapy was used to effectively treat 98% of patient bleeds with one or two doses. In the surgical clinical study of adolescents and adults, a single preoperative dose of REBINYN® provided a 100% success rate (defined by doctors as “excellent or good”) in bleeding control during surgery.
“Even with significant progress in the treatment of hemophilia B, there is an ongoing need for medicines that help patients reach and maintain high factor IX levels for a longer period of time,” said Guy Young, MD, Director of the Hemostasis and Thrombosis Program at Children's Hospital Los Angeles, and an investigator in the paradigm™ clinical trial program studying REBINYN®. “For my patients, an extended half-life treatment that can quickly control a bleed with just one or two doses is an important part of hemophilia B care.”
Source: PRNewswire, February 8, 2018