A Model-Based Framework to Inform the Dose Selection and Study Design of Emicizumab for Pediatric Patients With Hemophilia A.

ABSTRACT: Emicizumab is a bispecific antibody mimicking the cofactor function of activated coagulation factor VIII to prevent bleeds in patients with hemophilia A. The dose selection for the first-in-child phase III study of emicizumab was addressed by pediatric pharmacokinetic prediction using an adult/adolescent population pharmacokinetic model developed in phase I-I/II studies. The model was modified to incorporate functions describing the age-dependent increase in body weight (BW) with or without clearance maturation to account for the differences in emicizumab pharmacokinetics between adults/adolescents and children. A minimal dose anticipated to achieve in children the same target efficacious exposure as for adults/adolescents was identified when considering BW and clearance maturation. It was

SUBMITTER: Yoneyama K 

PROVIDER: S-EPMC9298840 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature