Nonacog beta pegol (N9-GP) is effective and well tolerated for the prevention and treatment of bleeding in children with haemophilia B

Results from two new interim analyses of the paradigm5 and paradigm6 children trials were presented at the 27th congress of the International Society on Thrombosis and Haemostasis (ISTH) in Melbourne, Australia. Nonacog beta pegol (N9-GP) demonstrated low annual bleeding rates and was well tolerated in children with haemophilia B, reinforcing the long-term safety and efficacy already established in previous trials.

In the five-year interim analysis of paradigm5, bleeding rates in previously treated children ( less then 12 years) with haemophilia B were low (median annualised bleeding rates [ABRs] were 0.66 overall, 0.0 for spontaneous bleeds and 0.47 for traumatic bleeds) and had declined after five years of treatment vs one year of treatment. 20% of children were bleed-free, and 64% had experienced no spontaneous bleeds throughout the trial. No children developed inhibitory antibodies and no safety signals were identified.The efficacy and safety profile of N9-GP is further supported by the first interim results of greater than 20 patients completing 50 exposure days (EDs) from paradigm6. Previously untreated children (<6 years) on weekly prophylaxis reported low bleeding rates and good bleed resolution with median ABRs of 0.0 for overall, spontaneous and traumatic bleeds. The incidence of inhibitory antibodies was within the expected range, with 2 out of 33 patients (6.1%) affected. No unexpected safety signals were seen.