Novo Nordisk’s recombinant coagulation factor IX nonacog beta pegol (N9-GP), under the U.S. trade name Rebinyn, has been approved by the U.S. Food and Drug Administration (FDA) to treat adults and children with hemophilia B. The company expects to launch this new treatment in the United States early in 2018.
The FDA decision was supported by positive effectiveness and safety results in four completed clinical trials (NCT00956345, NCT01333111, NCT01386528, NCT01395810) and preliminary results of two ongoing trials (NCT01467427, NCT02141074). The decision also follows a blood products advisory committee review.
“We would like to thank the patients who participated in the clinical studies that led to this decision. Thanks to their commitment, we are able to continue to provide new medicines for people with hemophilia,” Bill Breitenbach, Novo Nordisk vice president of biopharmaceuticals portfolio, said in a press release.
“We are committed to the hemophilia community and will continue on our path to bring this new extended half-life treatment to patients who need it,” Breitenbach said.
Rebinyn, given via intravenous injection, replaces the clotting factor IX that is missing in hemophilia B. The treatment was approved to be used as on-demand treatment to control bleeding episodes and for perioperative bleeding management.
The treatment is not for routine prophylaxis (preventive treatment) or for immune tolerance therapy in hemophilia B patients, according to approved prescribing information. The most common adverse reactions to treatment were itching and injection site reactions.
“We are excited about the approval of Rebinyn in the U.S., and we consider it an important expansion of the treatment options for patients with hemophilia B,” Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk, said in a press release.
“We are confident that Rebinyn will become an important tool for physicians to help patients manage their bleeds,” Thomsen said.
Novo Nordisk received a positive opinion in March 2017 from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), for the use of Refixia ― the trade name for N9-GP in Europe ― for prophylaxis and on-demand treatment of bleeding and surgical procedures in patients older than 12 with hemophilia B.