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

There are a number of pipeline/recently approved candidates in the hemophilia space that may make a significant impact in the market over the forecast period. Based on discussions with KOLs, we expect that bispecific antibodies will further extend their lead in the hemophilia A space, with Hemlibra 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 in the US for hemophilia A and B) and an initial reluctance from prescribers owing to uncertainty over their long-term efficacy and safety.