Novo Nordisk Files for Regulatory Approval Of Long-Acting Factor IX in EU for Treatment of Haemophilia B

Novo Nordisk (NYSE: NVO) today announced the submission to the European Medicines Agency of the Marketing Authorisation Application for the approval of long-acting factor IX, nonacog beta pegol. Nonacog beta pegol is a glycopegylated recombinant factor IX with a significantly improved pharmacokinetic (PK) profile, developed for patients with haemophilia B.Novo Nordisk expects to file the Biologics License Application (BLA) for nonacog beta pegol to the US Food and Drug Administration during first half of 2016.The filing of nonacog beta pegol is based on the results from the paradigm clinical trial programme, which involved 115 patients with severe or moderately severe haemophilia B. Nonacog beta pegol was found to be efficacious in routine prophylaxis, treatment of bleeding episodes and surgery for adults, adolescents and children. Furthermore, nonacog beta pegol appeared to be well-tolerated and no safety concerns were identified. Compared to standard factor IX products, nonacog beta pegol has a five times longer half-life. Patients in the paradigm study achieved a higher level of factor IX in the blood despite less frequent dosing of nonacog beta pegol. In the phase 3 trials, once-weekly administration of 40 IU/kg nonacog beta pegol maintained factor IX activity levels above 15%, reduced the median annualised bleeding rate (ABR) to 1.0 and showed a potential to prevent bleeds in target joints. Furthermore, these patients reported an improvement in quality of life during the trial. "With the regulatory filing of our long-acting factor IX, patients with haemophilia B are one step closer to having a new treatment option" said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk. "With its high factor activity level, less frequent dosing and very low ABRs, nonacog beta pegol has the potential to improve the quality of life for both patients and their families."