Pharmacokinetic properties of BAY 81-8973, a full-length recombinant factor VIII

• Published in: Haemophilia : the official journal of the World Federation of Hemophilia 2015-11, Vol.21 (6), p.766-771
• Main Authors: Shah, A., Delesen, H., Garger, S., Lalezari, S.
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aging - metabolism; Child; Child, Preschool; clinical trial; Ethnic Groups; Factor VIII - pharmacokinetics; Female; haemophilia A; half-life; Humans; Male; Middle Aged; pharmacokinetics; prophylaxis; recombinant FVIII; Recombinant Proteins - pharmacokinetics; Young Adult
• ISSN: 1351-8216; 1365-2516
• DOI: 10.1111/hae.12691

Introduction BAY 81‐8973 is a full‐length recombinant factor VIII (FVIII) with the same primary amino acid sequence as sucrose‐formulated recombinant FVIII (rFVIII‐FS) but is produced with advanced manufacturing technologies. Aim To analyse the pharmacokinetics (PK) of BAY 81‐8973 after single and multiple dosing across different age and ethnic groups in the LEOPOLD clinical trial programme. Methods The LEOPOLD trials enrolled patients with severe haemophilia A aged 12–65 years (LEOPOLD I and II) or ≤12 years (LEOPOLD Kids) with ≥150 (LEOPOLD I and II) or ≥50 (LEOPOLD Kids) exposure days to any FVIII product and no history of FVIII inhibitors. PK were assessed using chromogenic and one‐stage assays (only chromogenic assay for LEOPOLD Kids) after a single 50‐IU kg−1 dose of BAY 81‐8973 and, in a subset of patients in LEOPOLD I, after repeated dosing. Pharmacokinetic analyses were also performed based on age (18 to 65, 12 to