Results were presented from mentor, a phase 3 trial examining the efficacy and safety of a recombinant factor XIII (rFXIII) compound for the prevention of bleeds associated with congenital FXIII deficiency, a rare bleeding disorder with about 600–1,000 diagnosed patients worldwide.
In the trial, 41 patients were treated for one year, with rFXIII administered as a preventative, once-monthly replacement therapy for congenital FXIII deficiency. FXIII-deficient patients with no previous history of FXIII treatment were used as a control. Currently, the only treatment available is derived from human blood plasma, which carries an inherent risk of infections.
The trial results demonstrated that treatment with monthly recombinant FXIII injections significantly decreased the number of bleeding episodes requiring treatment compared to the historic control group. Over the course of the treatment period, a total of five bleeding episodes were observed in four patients. All five events were associated with trauma, and were not related to low FXIII activity levels in patients. Additionally, no thromboembolic events or fatal adverse events were reported.
“These data show the potential for rFXIII to become a safe and effective treatment option for patients who would otherwise use treatments at risk for contamination,” said Prof Aida Inbal of the Hemostasis Unit and Hematology Clinic in the Institute of Hematology at Rabin Medical Center in Tel Aviv, Israel. “We think this is an extremely important milestone in the development of a treatment that is not sourced from human plasma for patients suffering from congenital FXIII deficiency.”
Novo Nordisk plans to file for US Food and Drug Administration approval of the rFXIII in the first half of 2011.
Date: December 14, 2010
Source: Novo Nordisk A/S