Hemophilia | Niche and Rare Pharmacor | G7 | 2015

Hemophilia is a monogenic bleeding disorder occurring primarily in males. In hemophilia A and hemophilia B, factor VIII or factor IX (respectively) is either absent or deficient in function. Several factor replacement serums are currently approved to treat hemophilia A and B, but a comparatively large unmet need remains for hemophilia A patients because of disappointing factor half-life extensions, compounded by the higher diagnosed prevalence of hemophilia A vs B. Launch of the new longer-acting factor concentrates and agents with novel mechanisms of action, for both type A and B, offer a chance to achieve improvements in clinical outcomes and address compliance issues. Using primary research conducted with expert U.S. and European hemophilia specialists, this report provides a comprehensive analysis of the competitive landscape and market opportunity for hemophilia. It includes a comprehensive analysis of patient populations, current therapies and medical practices, unmet needs, and emerging therapies.