Novo Nordisk is seeking approval from the European Medicines Agency to market its long-acting haemophilia B therapy nonacog beta pegol.
The product is a glycopegylated recombinant factor IX with a significantly improved pharmacokinetic profile and a five-times longer half-life than standard forms of the blood-clotting drug.
The filing is based on the results from the paradigm clinical trial programme, which involved 115 patients with severe or moderately severe haemophilia B, a genetic condition caused by missing or defective factor IX. The drug was found to effective in routine prophylaxis, treatment of bleeding episodes and surgery for adults, adolescents and children, and was well-tolerated by patients, Novo said.
In the Phase III trials, once-weekly administration of 40 IU/kg nonacog beta pegol maintained factor IX activity levels above 15%, reduced the median annualised bleeding rate 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.”
Novo said it expects to file the drug in the US during first half of this year.