Europe’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion of nonacog beta pegol, recommending marketing authorization for prophylaxis and on-demand treatment of bleeding, as well as for surgical procedures, in adolescent (>12 years of age) and adult patients with hemophilia B.
Nonacog beta pegol is a recombinant coagulation factor IX that replaces missing factor IX in patients with deficiency. The agent is available as a powder and solvent utilized for injection (500 IU, 1000 IU, and 2000 IU).
The agency’s decision was based on results from a phase III trial of 115 children and adults with moderate to severe hemophilia B, in which nonacog beta pegol resolved nearly 95 percent of bleeds. The most common side effects of nonacog beta pegol included nausea, pruritus, fatigue, and injection-site reactions. The agent was designated as an orphan medical product in 2009.
Source: EMA Summary, March 23, 2017.