A Pharmacometric Approach to Substitute for a Conventional Dose-Finding Study in Rare Diseases: Example of Phase III Dose Selection for Emicizumab in Hemophilia A

• DOI: 10.1007/s40262-017-0616-3
• Publication Date: 2017-12-06T04:18:28Z
• Authors: Koichiro Yoneyama; Christophe Schmitt; Naoki Kotani; Gallia G. Levy; Ryu Kasai; Satofumi Iida; Midori Shima; Takehiko Kawanishi
• Keywords: Adult; Male; Factor VIII; Adolescent; Dose-Response Relationship, Drug; Hemorrhage; Antibodies, Monoclonal, Humanized; Hemophilia A; Models, Biological; 3. Good health; Young Adult; 03 medical and health sciences; Rare Diseases; 0302 clinical medicine; Antibodies, Bispecific; Humans; Original Research Article

Emicizumab (ACE910) is a bispecific antibody mimicking the cofactor function of activated coagulation factor VIII. In phase I-I/II studies, emicizumab reduced bleeding frequency in patients with severe hemophilia A, regardless presence VIII inhibitors, at once-weekly subcutaneous doses 0.3, 1, and 3 mg/kg.