DRG anticipates the next ten years to be a transitional time in the treatment of hemophilia A for patients with and without inhibitors. With the uptake of Roche / Chugai’s bispecific antibody Hemlibra and a diverse pipeline, both in terms of technology type and administration route, we forecast significant changes in disease management. 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-TFPIMAb, concizumab, and BioMarin’s gene therapy, BMN 270. Despite novel nonfactor hemophilia agents forecast to launch, we expect the factor concentrate market to remain competitive, and the high demand for factor concentrates to persist.
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