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FDA Approves Biologics License Application for Novo Nordisk’s Hemophilia B Product

June 2, 2017
FDA Approves Biologics License Application for Novo Nordisk’s Hemophilia B Product

Novo Nordisk recently announced that the US Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for REBINYN®, the company’s recombinant, GlycoPEGylated therapy for the treatment of adults and children with hemophilia B (factor IX deficiency). A BLA is a request to the FDA for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.

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® is indicated for on-demand treatment and control of bleeding episodes, and the perioperative management of bleeding in adults and children with hemophilia B. REBINYN® is not indicated for routine prophylaxis or for immune tolerance induction in patients with hemophilia B. The efficacy and safety evaluation was based on 115 patient participants across four clinical trials.

“We would like to thank the patients who participated in the clinical studies that led to this decision. Thanks to their commitment, we are able to continue to provide new medicines for people with hemophilia,” said Bill Breitenbach, Vice President, Biopharmaceuticals Portfolio, Novo Nordisk. “We are committed to the hemophilia community and will continue on our path to bring this new extended half-life treatment to patients who need it.”

Novo Nordisk expects to launch REBINYN® in the U.S. in the first half of 2018.

Source: Novo Nordisk press release dated May 31, 2017