Credit: Piotr Bodzek
The US Food and Drug Administration (FDA) has approved a recombinant factor VIIa product (NovoSeven® RT) for the treatment of bleeding episodes and for perioperative management in patients with Glanzmann’s Thrombasthenia (GT) who are refractory to platelet transfusions and may or may not have antibodies to platelets.
GT is a rare genetic bleeding disorder with limited treatment options. Patients with GT have a lifelong susceptibility toward bleeding episodes.
The condition, which affects 1 in 1 million people globally, occurs because certain surface proteins on platelets are missing or nonfunctional, significantly impacting the blood’s ability to form strong clots.
Patients with GT typically receive platelet transfusions when experiencing severe bleeding or when undergoing surgical procedures. However, some patients do not respond well, or at all, to platelet transfusions.
The FDA approved NovoSeven® RT for these patients based on evidence collected from the global Glanzmann’s Thrombasthenia Registry and the Hemostasis & Thrombosis Research Society Registry.
The data included 92 GT patients treated with NovoSeven® RT for 266 severe bleeding episodes and 77 GT patients treated with NovoSeven® RT for 160 surgical and other invasive procedures.
The treatment was successful in 94.4% of bleeding episodes and 99.4% of surgical procedures, based on a review of the data by independent hematology experts.
Among 140 patients treated for 518 bleeding episodes, surgeries, or traumatic injuries, the following adverse events were reported: deep vein thrombosis (n=1), headache (n=2), fever (n=2), nausea (n=1), and dyspnea (n=1).
NovoSeven® RT is also approved in the European Union for the treatment of bleeding episodes in patients with GT. The product is marketed by NovoNordisk.
