The CHMP, under the European Medicines Agency (EMA), recommends the granting of a marketing authorization in Europe for Refixia for the treatment and prophylaxis (preventive therapy) of bleeding in patients 12 years and older with hemophilia B (congenital factor IX deficiency).
Refixia was designated as an orphan medicinal product on 15 May 2009. According to an EMA press release, Refixia will be available as a powder and solvent for solution for injection (500 IU, 1000 IU and 2000 IU). Nonacog beta pegol, the active substance in Refixia, is a recombinant coagulation factor IX. It replaces the missing factor IX in patients with hemophilia B, helping the blood to clot and giving temporary bleeding control.
The recommendation of Refixia was based on the results from the phase 3 clinical trial programme, which enrolled 115 children and adults with severe or moderately severe hemophilia B.
In January 2016, Novo Nordisk reported that in these studies, Refixia was found to be effective in routine prophylaxis, treatment of bleeding episodes and surgery. The treatment was well-tolerated, with no safety concerns observed.
Compared to other factor IX products, Refixia was found to have five times longer half-life. Patients enrolled in the studies achieved a higher level of factor IX in their blood, even with less frequent dosings of Refixia.
The trials also found that administration of Refixia 40 IU/kg once per week, maintained patients’ factor IX activity levels above 15 percent, and reduced the median annualised bleeding rate (ABR) to 1.0. The treatment also showed potential in preventing joint bleeding, and in improving patients quality of life.
“We are excited about the positive opinion obtained for Refixia® and it represents a significant milestone in our efforts to expand the treatment options for patients with haemophilia,” Mads Krogsgaard Thomsen, executive vice president and chief scientific officer of Novo Nordisk said in a recent press release. “We believe Refixia® with its strong clinical profile provides haemophilia B patients better protection against bleeds, even into damaged joints, and an overall improved quality of life.”