Outside factors

This year could see the first regulatory submission from a coming wave of new therapies for hemophilia prophylaxis that do not simply replace missing clotting factors, which has been the standard for care for decades. These new products could enable a more personalized approach to treating subsets of patients based on disease severity and individual preference.

The most advanced is a mAb in Phase III testing that mimics the biological role of Factor VIII. Four earlier stage programs are focused on rebalancing the coagulation cascade (see “Next Wave in Hemophilia”)...