GTC Biotherapeutics (fka GTCB)

[DewDiligence]

FDA Approves NovoSeven in Acquired Hemophilia

[NovoSeven is already approved in most countries for hereditary hemophilia, which is also the first indication to be sought by the GTC-LFB collaboration. However, acquired hemophilia is an important market in its own right and one that GTC-LFB ought to be able to challenge in due course.]

http://biz.yahoo.com/prnews/061017/nytu152.html?.v=66

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NovoSeven® Becomes First and Only Recombinant Therapy for Treatment of Rare, Life-Threatening Bleeding Disorder

Tuesday October 17, 2:27 pm ET

FDA Approves Supplemental Use of NovoSeven(R) Coagulation Factor VIIa (Recombinant) for Treatment of Acquired Hemophilia

PRINCETON, N.J., Oct. 17 /PRNewswire/ -- The United States Food and Drug Administration (FDA) has approved a new indication for NovoSeven® Coagulation Factor VIIa (Recombinant), making it the first and only recombinant therapy approved for the treatment of acquired hemophilia, a rare and potentially fatal bleeding disorder. As a recombinant therapy, NovoSeven® is not plasma-derived and poses no risk of human viral transmission through its use.

The FDA approved NovoSeven® for treatment of bleeding episodes in patients with acquired hemophilia, and in the prevention of bleeding in surgical interventions or invasive procedures in patients with acquired hemophilia.

"Novo Nordisk is proud of our dedication to hemostasis research and development, especially for rare bleeding disorders. This acquired hemophilia indication is in line with our core commitment to provide innovative therapies to people who need them," said Martin Soeters, president of Novo Nordisk Pharmaceuticals, Inc.

NovoSeven® was first introduced in 1999, and is currently indicated for use in treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX and for treatment of bleeding episodes in patients with congenital Factor VII deficiency. It is also indicated for prevention of bleeding in surgical interventions or invasive procedures in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX and in patients with congenital Factor VII deficiency.

"The approval of NovoSeven for use in patients with acquired hemophilia offers an effective choice for treating this life-threatening disorder," said Craig M. Kessler, MD, Professor of Medicine and Pathology, Georgetown University Medical Center. "Its use will help physicians to meet the main objective in treating the disorder -- stopping the bleeding episode."

About Acquired Hemophilia

Acquired hemophilia appears in up to 4 persons per million per year. It disrupts the body's natural blood clotting process, causing spontaneous, uncontrolled bleeding to occur, most often, in the skin and soft tissues. It is often difficult to recognize and can be fatal in up to 22 percent of patients. Unlike classical hemophilia, acquired hemophilia is not inherited.

With acquired hemophilia, the body produces antibodies against its own Factor VIII or Factor IX blood clotting proteins that are essential to the formation of an effective blood clot. The disorder can occur in men or women (median age 64). Often, an underlying medical condition is involved. Associations with autoimmune conditions, cancer, use of certain drugs and pregnancy (although rare) have been recognized. In about half of all cases, there is no underlying cause.

About the Data

Pooled data analysis from the Compassionate Use Programs and Hemophilia and Thrombosis Research Society (HTRS) registry show that NovoSeven® demonstrated overall efficacy -- i.e., "effective" and "partially effective" outcomes -- in the majority (78 percent) of patients treated for bleeding episodes. This translates into 77 percent efficacy seen in the Compassionate Use Programs and 83 percent efficacy in the HTRS registry.

About NovoSeven®

NovoSeven® is an activated recombinant version of Factor VII which is functionally indistinguishable from naturally occurring activated Factor VII. The product is currently approved for treatment of bleeding episodes in congenital hemophilia patients with inhibitors to Factor VIII or Factor IX and in patients with acquired hemophilia; the prevention of bleeding in surgical interventions or invasive procedures in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX and in patients with acquired hemophilia; treatment of bleeding episodes in patients with congenital Factor VII deficiency; and prevention of bleeding in surgical interventions or invasive procedures in patients with congenital Factor VII deficiency.

NovoSeven® is produced in baby hamster kidney cells which have been genetically engineered to express recombinant Factor VII. Because no human albumin is added as a stabilizer, nor is any other human proteins used during manufacturing or added to the final product, there is no risk of human blood- borne pathogen transmission. For prescribing information, please visit www.novoseven-us.com.

Most common adverse events: pyrexia, hemorrhage, injection site reaction, arthralgia, headache, hypertension, hypotension, nausea, vomiting, pain, edema and rash. Patients with disseminated intravascular coagulation (DIC), advanced atherosclerotic disease, crush injury, septicemia, or concomitant treatment with activated or nonactivated prothrombin complex concentrates (aPCCs/PCCs) may have a potential risk of developing thrombotic events in association with NovoSeven® treatment. Contraindicated in patients with known hypersensitivity to NovoSeven® or mouse, hamster, or bovine protein. The risk of potential interaction between NovoSeven® and coagulation factor concentrates has not been evaluated. Simultaneous use of aPCCs/PCCs, should be avoided. Serious adverse events which may or may not have been related to the use of NovoSeven® in acquired hemophilia (10 of 139 patients in the Compassionate Use Program, HTRS registry, and the published literature) may include thrombotic serious adverse events. Serious adverse events which may or may not have been related to the use of NovoSeven® occurred in 14 of 298 patients with hemophilia A or B with inhibitors in the initial clinical program.

About Novo Nordisk

Novo Nordisk is a healthcare company and a world leader in diabetes care. The company has the broadest diabetes product portfolio in the industry, including the most advanced products within the area of insulin delivery systems. In addition, Novo Nordisk has a leading position within areas such as hemostasis management, growth hormone therapy and hormone replacement therapy. Novo Nordisk manufactures and markets pharmaceutical products and services that make a significant difference to patients, the medical profession and society. With headquarters in Denmark, Novo Nordisk employs more than 22,750 employees in 79 countries, and markets its products in 179 countries. For more information, please visit www.novonordisk-us.com.
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