Nothing short of a revolution: Novel extended half-life factor VIII replacement products and non-replacement agents reshape the treatment landscape in hemophilia A.

ABSTRACT

Hemophilia A, an X-linked genetic disorder, is characterized by a deficiency or dysfunction of clotting Factor VIII. The treatment landscape has substantially changed by introducing novel extended half-life factor VIII (EHL-FVIII) replacement therapies such as efanesoctocog Alfa and non-factor replacement therapy such as emicizumab. These agents signal a shift from treatments requiring multiple weekly infusions to advanced therapies with long half-lives, offering superior protection against bleeding and improving patient adherence and quality of life. While EHL-FVIII treatment might lead to inhibitor development in some patients, non-factor replacement therapy carries thrombotic risks. Therefore, ongoing research and the generation of robust clinical evidence remain vital to guide the selection of optimal and cost-effective first-line therapies for hemophilia A patients.