Novo Nordisk files for regulatory approval in the US of long-acting factor IX for the treatment of haemophilia B

Bagsværd, Denmark, 16 May 2016 - Novo Nordisk today announced the submission to the US Food and Drug Administration (FDA) of the Biologics License 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.
 
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 circulation 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 a significant improvement in quality of life during the trial.
 
"With the regulatory filings in 2016 of our long-acting factor IX, people with haemophilia B in both the EU and the US 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 reduced bleeding rate, nonacog beta pegol has the potential to improve the quality of life for both patients and their families."
 

About nonacog beta pegol

Nonacog beta pegol is a factor IX molecule with an extended half-life intended 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, already proven safe and efficacious in haemophilia A and other therapeutic areas.  

 

About the paradigm clinical programme

The paradigm clinical trial programme for nonacog beta pegol enrolled children and adults with severe or moderately severe haemophilia B. A total of 115 previously treated patients with a total of more than 10,625 exposure days for up to 3.3 years of treatment with nonacog beta pegol.