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
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