An arm of the European Medicines Agency (EMA) known as the Committee for Medicinal Products for Human Use (CHMP) has recommended approving Refixin for preventing and treating patients 12 years and older with hemophilia B, or congenital factor IX deficiency. The European Commission will make the final decision on approval.
The EU designated Refixia (nonacog beta pegol, N9-GP) an orphan drug on May 15, 2009. The EMA said in a press release that the new recommendation covers Refixia as a powder and solvent for injection. The doses it covers include 500 IU, 1000 IU and 2000 IU.
Nonacog beta pegol, the active ingredient 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 providing temporary bleeding control.
CHMP’s Refixia recommendation was based on the results of a Phase 3 clinical trial that enrolled 115 children and adults with moderately severe to severe hemophilia B.
Novo Nordisk reported in January 2016 that trial investigators found Refixia effective in preventing and treating bleeding episodes, and controlling bleeding in surgery. The treatment was well-tolerated, with no safety problems, the investigators said.
Refixia’s half-life was five times longer than that of other factor IX products, the trial results showed. Participants achieved a higher level of factor IX in their blood than with other products, even with less frequent doses of Refixia. A drug’s half-life is the amount of time it takes for its effectiveness to drop in half.
The trial also found that 40 IU/kg of Refixia once per week kept patients’ factor IX levels above 15 percent, and reduced their median annualized bleeding rate (ABR) to 1.0. In addition, the treatment showed potential in preventing joint bleeding, and in improving patients’ quality of life.
“We are excited about the positive opinion obtained for Refixia, Mads Krogsgaard Thomsen, executive vice president and chief scientific officer of Novo Nordisk, said in a press release. It represents a significant milestone in our efforts to expand the treatment options for patients with haemophilia. We believe Refixia with its strong clinical profile provides hemophilia B patients better protection against bleeds, even into damaged joints, and an overall improved quality of life.”
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