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

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
DOI: 10.1007/s40262-017-0616-3 Publication Date: 2017-12-06T04:18:28Z
ABSTRACT
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.
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