FDA Approves Novo Nordisk A/S's NovoSeven® RT To Treat Glanzmann's Thrombasthenia (GT) With Refractoriness

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

• Serious blood clots that form in veins and arteries with the use of NovoSeven^® RT have been reported.
• Your healthcare provider should discuss the risks and explain the signs and symptoms of blood clots to you. Some signs of a blood clot may include pain, swelling, warmth, redness, or a lump in your legs or arms, chest pain, or sudden severe headache and/or loss of consciousness or function.
• Your healthcare provider should monitor you for blood clots during treatment with NovoSeven^® RT.

Warnings and Precautions

• NovoSeven^® RT should be used with caution if you have an increased risk for blood clots, such as with disseminated intravascular coagulation (DIC), clogged arteries, crush injury, septicemia (an infection in the blood), uncontrolled bleeding after giving birth, history of heart disease, liver disease, limited movement following surgery, in elderly people, in newborns, or if you are taking aPCCs/PCCs (activated or nonactivated prothrombin complex concentrates) with NovoSeven^® RT.
• Allergic reactions, including serious whole body allergic reactions, have been reported with NovoSeven^® RT. Tell your healthcare provider if you are allergic to NovoSeven^® RT, any of its ingredients, or mice, hamsters, or cows. If you think you are having an allergic reaction, call your healthcare provider right away. Some signs of allergic reaction may include shortness of breath, rash, itching, redness of the skin, and fainting/dizziness.
• People with Factor VII deficiency should be monitored by their healthcare provider for antibodies, which can cause NovoSeven^® RT to stop working properly.

Side Effects

• The most common and serious side effects are blood clots.
• Tell your healthcare provider about any side effects that bother you or do not go away.

Use with Other Drugs

• Blood clots may occur if NovoSeven^® RT is given with Coagulation Factor XIII (13).

Please visit www.novosevenrt.com for full Prescribing Information.

About Novo Nordisk
Headquartered in Denmark, Novo Nordisk is a global healthcare company with 90 years of innovation and leadership in diabetes care. The company also has leading positions within hemophilia care, growth hormone therapy and hormone replacement therapy. For more information, visit www.novonordisk-us.com or follow our news in the US on Twitter: @novonordiskus.

[i] National Organization for Rare Disorders. Glanzmann's thrombasthenia. http://www.rarediseases.org/rare-disease-information/rare-diseases/byID/156/viewAbstract.  Accessed May 7, 2014.
[ii] Poon M. Clinical use of recombinant human activated factor VII (rFVIIa) in the prevention and treatment of bleeding episodes in patients with Glanzmann's thrombasthenia. Vascular Health and Risk Management. 2007;3(5): 655-664.
[iii] Nurden A. Glanzmann's thrombasthenia. Orphanet J Rare Dis. 2006;1:10.
[iv] DiMinno G, et al. Glanzmann's thrombasthenia (defective platelet integrin allb-b3): proposals for management between evidence and open issues. Thromb Haemost. 2009;102:1157-1164.
[v] NovoSeven^® RT (Coagulation Factor VIIa [Recombinant]) Prescribing Information. July 2014.

© 2014 Novo Nordisk                  All rights reserved.                  0514-00021424-1                  July 2014

SOURCE Novo Nordisk