The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A.

ABSTRACT: N8-GP (turoctocog alfa pegol) is a recombinant, glycoPEGylated, extended half-life, factor VIII replacement product. Here, we examined the immunogenicity, safety, and efficacy of N8-GP in previously untreated patients (PUPs). pathfinder6 is an ongoing, open-label, phase 3 trial that enrolled PUPs with severe hemophilia A and were aged <6 years. The primary end point was the incidence of factor VIII inhibitors (≥0.6 Bethesda units [BU]). Eighty patients received ≥1 N8-GP dose and were included in this analysis; ≥50 patients had ≥50 exposure days to N8-GP. The inhibitor incidence was 29.9% (14.9% high-titer [>5 BU]). Sixty-five patients received N8-GP prophylaxis for an average of 2.17 years with a median annualized bleeding rate (interquartile range) of 1.42 (0.76; 3.13) and a 90.5% hemosta

SUBMITTER: Male C 

PROVIDER: S-EPMC9979760 | biostudies-literature | 2023 Feb

REPOSITORIES: biostudies-literature