Novo Nordisk announced that the Committee for Medicinal Products for Human Use (CHMP), under the European Medicines Agency (EMA), adopted a positive opinion for the use of Refixia (nonacog beta pegol, N9-GP), recommending marketing authorisation for the treatment of adolescents and adults with hemophilia B.
The CHMP recommends Refixia, the brand name for nonacog beta pegol, N9-GP, to be indicated for prophylaxis and on-demand treatment of bleeding as well as for surgical procedures in adolescent (>12 years of age) and adult patients with haemophilia B (congenital factor IX deficiency). The recommendation is based on the results from the paradigm clinical trial programme, where 115 previously treated children and adults with haemophilia B were treated with Refixia.
“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,” said Mads Krogsgaard Thomsen, executive vice president and chief scientific officer of Novo Nordisk. “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.”
Refixia (nonacog beta pegol, N9-GP) is an extended half-life factor IX molecule for replacement therapy in patients with haemophilia B. Glycopegylation, the prolongation technology used for the half-life extension, is a novel approach in haemophilia B. Pegylated products have been approved in haemophilia A and other therapeutic areas. The review of Refixia® was based on the paradigm programme, a phase 3 clinical programme enrolling children and adults with severe or moderately severe haemophilia B. In the programme, 115 previously treated patients had a total of more than 8,800 exposure days for up to 2.7 years of treatment with Refixia.