FDA Approves NovoSeven® RT for Glanzmann’s Thrombasthenia

On July 7, 2014, the US Food and Drug Administration (FDA) approved NovoSeven® RT, a recombinant factor VIIa product manufactured by NovoNordisk, as the first recombinant treatment for Glanzmann’s Thrombasthenia (GT). The approved indication is for bleeding episodes and perioperative management for patients in whom platelet transfusions, with or without antibodies to platelets, is ineffective.
GT is a platelet function disorder that is very rare, occurring in about one in one million people. People who inherit the condition have defective genes for the glycoprotein llb/llla, which allows platelets to form a plug at the site of an injury.
Symptoms are similar to severe hemophilia–easy bruising, nose bleeds, oral bleeding, heavy and/or prolonged menstrual bleeding (menorrhagia) and hemorrhaging after childbirth, surgery or circumcision. In some people the condition can cause urinary or gastrointestinal tract bleeding.
Previously, patients were treated with platelet transfusions. However, some experienced refractoriness, the inability to achieve the needed level of platelets. Some reasons for this reaction include the development of antibodies to the infused platelets, signaling an immune response, or fever or recent chemotherapy administration.
The FDA approval is based on data from two registries: the global Glanzmann’s Thrombasthenia Registry (GTR), which includes 218 patients with 1,073 bleeding and surgical events, and the Hemostasis & Thrombosis Research Society Registry, which includes 7 patients with 23 bleeding episodes.
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. The new drug was successful in 94.4% of bleeding episodes and 99.4% of surgical procedures, based on review of data by independent hematology experts. Side effects occurred in a handful of subjects, and included deep-vein thrombosis, fever and headache. This data demonstrated that NovoSeven® RT was safe and effective in the treatment of bleeding and for prevention of bleeding during surgical procedures in patients with GT.
“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.”
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