FDA approves NovoSeven RT for Glanzmann's thrombasthenia

FDA approved coagulation factor VIIa [recombinant] (NovoSeven RT, Novo Nordisk), for the treatment of Glanzmann’s thrombasthenia, a rare genetic bleeding disorder.

According to the Novo Nordisk, NovoSeven RT is the first recombinant treatment for bleeding episodes and perioperative management in patients with Glanzmann’s thrombasthenia with refractoriness to platelet transfusions, with or without antibodies to platelets.

NovoSeven RT is indicated for the 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. In addition, it is also indicated for the treatment of bleeding and prevention of bleeding for surgeries and procedures in adults with acquired hemophilia.

Glanzmann’s thrombasthenia affects 1 million people worldwide, and patients have a lifelong susceptibility toward bleeding episodes, including frequent, severe, and potentially life-threatening hemorrhage. Platelet transfusions are usually administered to control severe bleeding or when surgical procedures are required, but some patients do not respond well or at all to platelet transfusions.

NovoSeven RT was approved based on evidence collected from the global Glanzmann's Thrombasthenia Registry (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 µg/kg) to treat bleeding as well as preventing bleeding during surgical procedures.

Treatment with this agent was successful in 94.4% of bleeding episodes and 99.4% 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).