Novo Nordisk has announced that their hemophilia B treatment, Rebinyn, is now available in the United States.
is a serious, chronic bleeding disorder that slows the blood clotting process in a patient due to a lack of clotting Factor IX protein. This results in prolonged periods of bleeding or oozing after injury or surgery.
Patients with severe cases can experience excessive bleeding from small or minor cuts. Complications can arise from bleeding into the joints, muscles, brain or other internal organs. Approximately 5,000 people in the U.S. are diagnosed with hemophilia B.
The FDA approved Rebinyn in May 2017.
Rebinyn treats adults and children with hemophilia B through an extended half-life injectable medicine that controls bleeding. The therapy can be used when injuries or cuts occur or to manage bleeding during surgery, however, Rebinyn is not to be used regularly as routine prophylaxis in patients.
The technology that extends the half-life of factor in the blood is called GlycoPEGylation. This innovative advancement allows Rebinyn to be kept in the bloodstream longer to stop bleeding without the need for extra doses.
"We're excited to make this new treatment option available to the hemophilia community," said Pia D' Urbano, Corporate Vice President, Biopharmaceuticals, Novo Nordisk Inc. in a press release
. "The introduction of Rebinyn®
energizes us to build on our rich legacy of making innovative medicines available for people with rare bleeding disorders."
During Phase 3 studies, one dose of Rebinyn in adults elevated Factor IX activity above baseline levels by 94% and sustain average levels of factor 17% one week after the injection. An 83-hour average half-life was achieved in adults after receiving a single infusion of Rebinyn.
Rebinyn was able to treat 98% of patient bleeds in a recent adolescent and adult clinical trial. In another clinical trial, the drug had a 100% success rate among adolescents and adults in controlling bleeding 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," shared Guy Young
, M.D., 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."