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Background  N8‐ GP  is an extended half‐life recombinant factor VIII developed for prophylaxis and treatment of bleeds in patients with hemophilia A.    Objective  To assess pharmacokinetic ( PK ) characteristics of N8‐ GP  in previously treated patients with severe hemophilia A, model the time spent at hemophilia thresholds of ≥1 and ≤5 IU/dL (moderate) or >5 IU/dL (mild)  FVIII  levels during N8‐ GP  prophylaxis, and investigate the relationship between N8‐ GP  half‐life and von Willebrand factor ( vWF ).    Methods   PK  assessments were obtained from patients with severe hemophilia A ( FVIII  < 1 IU/dL) participating in 4 clinical trials: pathfinder 1 (20‐60 years); pathfinder 2 (12‐17 and ≥18 years); pathfinder 5 (0‐11 years), and pathfinder 7 (25‐71 years). All  PK  profiles were assessed after washout and considered single‐dose  PK  profiles. Pre‐ and postdose  FVIII  activity at steady state was measured at all visits.    Results  From 69 patients, 108  PK  profiles of N8‐ GP  50 IU/kg were assessed. Adults/adolescents received 50 IU/kg every 4 days, achieving mean trough levels of 3.0 IU/dL (95%  confidence interval,  2.6‐3.5, adults) and 2.7 IU/dL (1.8‐4.0, adolescents). Children received 60 IU/kg twice weekly, leading to mean trough levels of 1.2 IU/dL (0.8‐1.6, 0‐ to 5‐year‐olds) and 2.0 IU/dL (1.5‐2.7, 6‐ to 11‐year‐olds).  PK  modeling predicted children dosed every 3 days and adults/adolescents dosed every 3 to 4 days would maintain  FVIII  levels >5 and >1 IU/dL for >80% and 100% of the time, respectively. N8‐ GP  half‐life correlated linearly with von Willebrand factor levels in adults/adolescents, less in children.    Conclusions  Prophylaxis with fixed intervals (Q4D/twice weekly) and fixed weight‐based dosing (50/60 IU/kg) ensured >1 IU/dL  FVIII  trough levels in both adults and children.

Fixed doses of N8‐ GP prophylaxis maintain moderate‐to‐mild factor VIII levels in the majority of patients with severe hemophilia A