Novo Nordisk has filed a recombinant factor XIII compound with regulators in the USA as a treatment for a bleeding disorder that affects one in two million people.
The Danish drugmaker has submitted a Biologic License Application to the US Food and Drug Administration for rFXIII to treat "the vast majority of patients with congenital factor XIII deficiency", an inherited bleeding disorder. Currently, patients are treated with products derived from human blood plasma "which carry an inherent risk of contamination", Novo noted, while its treatment is genetically engineered and reduces the potential risk of transmitting blood-borne illnesses.
Only 600 patients worldwide have been diagnosed with the disorder caused by a lack of the protein clotting factor XIII, and Eddie Williams, vice president of biopharmaceuticals at Novo, said the development of a treatment that does not depend on blood products is an important step.
The submission to the FDA is based on a one-year 41-patient Phase III trial, where rFXIII was administered as a preventive, once-monthly replacement therapy. The company will file its marketing authorisation application to the European Medicines Agency in the second quarter.