DRG expects the next ten years to be transitional for patients with hemophilia A, with and without inhibitors, as both the number of therapy options and size of the hemophilia market increase. With the emergence of Roche / Chugai’s bispecific antibody Hemlibra and a diverse pipeline, both in terms of technology type and administration route, we forecast a significant change in approach to disease management for many patients. The R&D emphasis has shifted to nonfactor treatment approaches to restore coagulation. Examples include Sanofi’s anti-ATIII siRNA fitusiran, Novo Nordisk’s anti-TFPI MAb concizumab, and BioMarin’s gene therapy BMN 270. Despite novel nonfactor hemophilia agents forecast to launch, we expect the demand for factor-based concentrates to persist.
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