Novo Nordisk files BLA for first recombinant treatment for FXIII in USA

The US unit of Danish insulin giant Novo Nordisk (NVO: N) has submitted a Biologic License Application (BLA) to the Food and Drug Administration requesting approval of a recombinant Factor XIII compound (rFXIII) for the vast majority of patients with congenital factor XIII (FXIII) deficiency, an inherited bleeding disorder. The company anticipates filing its marketing authorization application to the European Medicines Agency in the second quarter of 2011.

Currently, FXIII deficient patients are treated with products derived from human blood plasma, which carry an inherent risk of contamination, says Novo Nordisk. 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. With an estimated 600 diagnosed patients worldwide, FXIII deficiency is considered one of the rarest bleeding disorders.