Special Report: Do Emerging Therapies Offer Better Treatment Options for Hemophilia Patients?

Introduction:

A robust pipeline offers potential new approaches to improve the existing therapies used for treating hemophilia, a bleeding disorder that is caused mainly by the deficiency of clotting factor VIII and IX. The disease is treated by giving intravenous transfusions of the deficient factor, but a strong need remains for the development of new factors that would eliminate or reduce inhibitor production against the factor replacement therapy. Interviewed experts eagerly await the approval of emerging therapies as the leading companies in this field pursue further clinical development of their late-stage products.

Questions Answered in This Report:

• Several recombinant products have entered the hemophilia market in the past five years. These recombinant agents are preferred over human plasma products. What are the sales of the top recombinant products for factor VIII replacement therapy? Which of these agents has the largest sales and why? What are these agents’ shortcomings, according to interviewed experts?
• Several emerging agents are in the pipeline. What improvement do they offer over the current factor replacement therapies? If approved, how are they likely to affect the existing treatment of hemophilia patients? What do thought-leading experts say about these agents?
• A strong unmet need remains for new factors that do not provoke inhibitor production, as current replacements factors do, or at least offer reduced problems with inhibitor production. What are the other unmet needs identified by interviewed experts? How many of these unmet needs will emerging agents address? Which company has the maximum number of promising agents in development?