Hemophilia A patients are a heterogeneous cohort. The tendency to bleed is highly individual, resulting in a complex management landscape. Two core therapeutic approaches are employed: (1) prophylaxis, where the aim is to prevent clinically meaningful bleeds through maintaining factor VIII (FVIII) levels, and (2) on-demand treatment, where drugs are utilized at the time of a significant bleed. Various similar FVIII replacement therapies are available (e.g., Takeda’s Advate, Sanofi’s Eloctate). However, some patients develop inhibitors (alloantibodies) that can reduce the effectiveness of FVIII replacement. The launch of Roche’s novel subcutaneous therapy Hemlibra has impacted the hemophilia A +/- inhibitor markets in differing ways. In the noninhibitor space, compelling reasons exist for many hemophilia A patients to remain on FVIII replacement therapies. In contrast, Hemlibra has dramatically altered treatment dynamics in the inhibitor space and has spurred much discussion regarding its merits in the management of newly diagnosed inhibitor patients.
Geographies: United States.
Primary research: Survey of 100 hematologists.
Key drugs covered: Advate, Adynovate, Afstyla, Eloctate, FEIBA, Hemlibra, Jivi, Kogenate, Kovaltry, Novoeight, NovoSeven, Nuwiq.
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