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
AUTHORS (8)
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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