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.


  • How will physicians treating hemophilia A differentiate between the various emerging therapies, and which will gain the greatest market and patient share in an increasingly stringent regulatory environment?
  • What clinical roles will Hemlibra, fitusiran, concizumab, and gene therapy play in the evolving hemophilia A treatment landscape?
  • How will the management of patients with inhibitors change when new treatment options become available? How will Hemlibra’s head start on its competitors play out over the forecast period?
  • How will the factor VIII concentrate market evolve? How will nonfactor treatment options impact the multitude of brands vying for patient share in this space?


Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

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