The U.S. Food and Drug Administration (FDA) has approved the biologics license application for a modified recombinant coagulation factor IX (FIX) nonacog beta pegol, for the treatment of adults and children with hemophilia B.
Nonacog beta pegol is indicated for on-demand treatment and control of bleeding in patients with clotting FIX deficiency. It is not intended for prophylactic treatment or immune tolerance induction.
According to a briefing prepared by the coagulation factor’s sponsor for the FDA’s Blood Products Advisory Committee, nonacog beta pegol increases the “incremental recovery … of FIX activity compared to all currently approved FIX products.”
The decision was based on data from four paradigm™ trials of patients with severe or moderate hemophilia B (FIX ≤2%) – two are still ongoing, including one extension trial and one with previously untreated patients. When used as on-demand treatment for controlling bleeding episodes, nonacog beta pegol 40 IU/kg once-weekly successfully treated 93.2 percent of bleeds, including 97 percent of bleeds in adolescents and adults and 93 percent of bleeds in children.
Researchers found no unexpected clinical safety concerns with nonacog beta pegol, though the FDA briefing noted that rates of neurologic AEs will need to be monitored closely.
Sources: Novo Nordisk press release, May 31, 2017; FDA Blood Products Advisory Committee briefing document, April 4, 2017.