PLAINSBORO, N.J., July 7, 2014 /PRNewswire/ -- Novo Nordisk today announced the US Food and Drug Administration (FDA) has approved NovoSeven(®) RT (Coagulation Factor VIIa [Recombinant]) as the first recombinant treatment for bleeding episodes and perioperative management in patients with Glanzmann's Thrombasthenia (GT) with refractoriness to platelet transfusions, with or without antibodies to platelets. Glanzmann's Thrombasthenia is a rare genetic bleeding disorder with limited treatment options. The safety and effectiveness of NovoSeven(®) RT were demonstrated in the treatment of severe bleeding episodes and the perioperative management for major and minor surgical procedures for this specific patient population.
Patients with GT have a lifelong susceptibility toward bleeding episodes, including frequent, severe, and potentially life-threatening hemorrhages.[i] The condition, which affects one in 1 million people globally,[ii] occurs because certain surface proteins on platelets are missing or do not work, significantly impacting the blood's ability to form strong clots.[iii] Patients with GT may be administered platelet transfusions when experiencing severe bleeding or when surgical procedures are required. However, some patients do not respond well or at all to platelet transfusions (called refractoriness).[iv]
"Novo Nordisk is proud to build on our strong heritage of pioneering new therapies and indications for the bleeding disorders community by offering the only recombinant treatment for Glanzmann's Thrombasthenia," said Eddie Williams, Senior Vice President, BioPharmaceuticals, Novo Nordisk. "We're committed to keeping patients at the forefront of all we do, and this approval allows us to expand on this commitment."
The FDA approved NovoSeven(®) RT for the treatment of GT based upon evidence collected from the global Glanzmann's Thrombasthenia Registry (GTR) (218 patients with 1,073 bleeding and surgical events) and the Hemostasis & Thrombosis Research Society Registry (7 patients with 23 bleeding episodes). The data supported the effectiveness and safety of NovoSeven(®) RT (90 mcg/kg) in the treatment of bleeding and for the prevention of bleeding during surgical procedures in patients with GT.
Overall, the GTR collected data from 92 patients treated with NovoSeven(®) RT for 266 severe bleeding episodes and 77 patients treated with NovoSeven(®) RT for 160 surgical and other invasive procedures. Treatment with NovoSeven(®) RT was successful in 94.4 percent of bleeding episodes and 99.4 percent of surgical procedures, based upon review of data by independent hematology experts. Of 140 patients treated for 518 bleeding episodes, surgeries, or traumatic injuries, the following adverse reactions were reported: deep vein thrombosis (n=1), headache (n=2), fever (n=2), nausea (n=1), and dyspnea (n=1).[v]
NovoSeven(®) RT also is approved in the European Union for the treatment of bleeding episodes in patients with GT.
Indications and Usage
NovoSeven(®) RT (Coagulation Factor VIIa [Recombinant]) is used for:
-- Treatment of bleeding and prevention of bleeding for surgeries and procedures in adults and children with hemophilia A or B with inhibitors, congenital Factor VII (FVII) deficiency, and people with Glanzmann's thrombasthenia who have a decreased or absent response to platelet transfusions -- Treatment of bleeding and prevention of bleeding for surgeries and procedures in adults with acquired hemophilia
Important Safety Information
WARNING: BLOOD CLOTS