Once‐weekly prophylaxis with glycoPEGylated recombinant factor VIII (N8‐GP) in severe haemophilia A: Safety and efficacy results from pathfinder 2 (randomized phase III trial)

• DOI: 10.1111/hae.13712
• Publication Date: 2019-02-28T16:54:30Z
• Authors: Nicola Curry; Canan Albayrak; Miguel Escobar; Pål Andre Holme; Susan Kearney; Robert Klamroth; Mudi Misgav; Claude Négrier; Allison Wheeler; Elena Santagostino; Midori Shima; Andrea Landorph; Sidsel Marie Tønder; Steven R. Lentz
• Keywords: FVIII; safety; Adult; Male; Factor VIII; efficacy; 610; haemophilia A; Hemorrhage; Hemophilia A; Drug Administration Schedule; Polyethylene Glycols; 3. Good health; N8-GP; 03 medical and health sciences; once-weekly prophylaxis; 0302 clinical medicine; Humans; Female; ORIGINAL ARTICLES; Safety

Introduction Turoctocog alfa pegol (N8‐GP) is a site‐specific, 40 kDa glycoPEGylated recombinant factor VIII (FVIII) product with an extended half‐life. The comprehensive main phase of the pivotal pathfinder 2 trial showed N8‐GP dosed every 4 days (Q4D) provided favourable safety and efficacy for preventing bleeds in 175 patients haemophilia A. Aim methods We investigated prophylaxis when administered weekly (Q7D) 24 weeks to low bleeding rates extension trial. Patients (≥12 years) ≤2 during preceding 6 months were eligible randomization receive 50 IU/kg Q4D or 75 Q7D. Safety endpoints incidence FVIII inhibitors annualized rate (ABR), respectively. Results Fifty‐five 143 (38.5%) on who continued into randomized (n = 17) Q7D 38). Nine cohort reverted Q4D. No detected. In both cohorts, >50% experienced no bleeds. Median ABR overall, joint, spontaneous, traumatic muscle was 0.00 cohorts. Overall estimated success treating episodes 87.5%; 94.7% controlled injections. Conclusions Weekly well tolerated efficacious may benefit selected “low bleeder”