Therapeutic strategies for hemophilia were revolutionized after the introduction of replacement factors during the late 1990s and early 2000s, which were followed by extended half-life recombinant factor products (EHLs) over the past decade. Currently, the hemophilia market is undergoing a third revolution, with an anticipated shift towards alternative coagulation promoters (ACPs) and gene therapy. 

There are a number of pipeline/recently approved candidates in the hemophilia space which may make a significant impact in the market over the forecast period. Based on discussions with KOLs, we expect that Roche’s Hemlibra will further extend its lead in the hemophilia space, having captured market leader status from Takeda’s Advate in 2019. Gene therapies are expected to have initially muted uptake because of a high upfront cost for payers ($2.9m–$3.5m US range for hemophilia A and B) and an initial reluctance from prescribers owing to uncertainty over their long-term efficacy and safety. 

This Datamonitor Healthcare report contains a Disease Analysis module.