Novo Nordisk (NJ) Submits First Recombinant Treatment for Factor XIII Deficiency for U.S. Regulatory Approval

PRINCETON, N.J., Feb. 23, 2011 /PRNewswire/ -- Novo Nordisk announced today that a Biologic License Application (BLA) has been submitted to the U.S. Food and Drug Administration (FDA) requesting the approval of a recombinant factor XIII compound (rFXIII) for the vast majority of patients with congenital factor XIII (FXIII) deficiency, an inherited bleeding disorder(1). The company anticipates filing its marketing authorization application to the European Medicines Agency in Q2 2011.

Currently, FXIII deficient patients are treated with products derived from human blood plasma(2), which carry an inherent risk of contamination. Treatments created using recombinant technology are genetically engineered and not sourced from human blood or plasma. The use of this technology in the development of treatments helps to reduce the potential risk of transmitting blood-borne illnesses.

Patients with congenital FXIII deficiency have a lifelong susceptibility towards bleeding problems, including spontaneous intracranial hemorrhage. Caused by a lack of the protein clotting factor XIII, FXIII congenital deficiency has a prevalence of one case per two million people(2). With an estimated 600 diagnosed patients worldwide, FXIII deficiency is considered one of the rarest bleeding disorders(3).

“Novo Nordisk is committed to serving the bleeding disorder community, and this submission marks a major milestone in the expansion of our product offerings to this patient group,” said Eddie Williams, Vice President of Biopharmaceuticals at Novo Nordisk. “The development of a treatment that does not depend on blood products is an important step for the hundreds of patients affected worldwide by congenital FXIII deficiency.”

Positive results from a phase 3 trial examining the efficacy and safety of rFXIII for the prevention of bleeds associated with congenital FXIII deficiency showed that when compared to a historic control group of individuals who did not receive routine FXIII infusions, preventive treatment with monthly recombinant FXIII injections significantly decreased the number of bleeding episodes requiring treatment. These data were presented at the American Society of Hematology (ASH) meeting in December 2010, and marked the first completed phase 3 study conducted to study the use of a recombinant FXIII treatment to prevent bleeding episodes in congenital FXIII deficiency patients.

In the trial, 41 patients were observed over one year, with rFXIII administered as a preventive, once-monthly replacement therapy for FXIII congenital deficiency. Over the course of the treatment period, a total of five bleeding episodes, all associated with trauma and requiring additional FXIII treatment, were observed in four patients. Additionally, no thromboembolic events or fatal adverse events were reported.

About Novo Nordisk

Novo Nordisk is a global healthcare company with more than 87 years of innovation and leadership in diabetes care. The company also has leading positions within hemophilia care, growth hormone therapy and hormone therapy for women. Novo Nordisk’s business is driven by the Triple Bottom Line: a commitment to social responsibility to employees and customers, environmental soundness and economic success. Headquartered in Denmark, Novo Nordisk employs more than 30,000 employees in 81 countries, and markets its products in 179 countries. Novo Nordisk’s B shares are listed on the stock exchange in Copenhagen. Its ADRs are listed on the New York Stock Exchange (NVO). For more information, visit novonordisk-us.com.

(1) Bolton-Maggs PH, Perry DJ, Chalmers EA, et al. The rare coagulation disorders--review with guidelines for management from the United Kingdom Haemophilia Centre Doctors’ Organisation. Haemophilia 2004; 10: 593628.

(2) Karimi M et al. Factor XIII deficiency. Semin Thromb Haemost 2009;35(4): 426-38.

(3) National Hemophilia Foundation. Factor XIII. http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=190&contentid=58&rptname=bleeding. Accessed February 8, 2011.

SOURCE Novo Nordisk