Clarivate expects the next 10 years to be transitional for patients with hemophilia A (with and without inhibitors) owing to the entry of several novel therapeutic options into the market. 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 the approach to disease management of many patients. The emphasis in R&D has shifted away from factor-VIII-replacement-based approaches to new mechanisms aimed at restoring coagulation, including gene therapies. These new therapies include Sanofi’s fitusiran and BIVV001, Novo Nordisk’s concizumab, Pfizer’s marstacimab, and BioMarin’s Roctavian. Despite the various novel nonfactor hemophilia agents forecast to launch, we expect the demand for factor-based concentrates to persist.
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