"Ultra-Long FVIII: A Major Step Forward to a Hemophilia-Free Mind"

A remarkable step forward in the treatment of hemophilia A has recently been achieved with the development of an Ultra-Long modified FVIII. Leveraging expertise gained with fusion to immunoglobulin Fc fragments, disconnecting FVIII from endogenous von Willebrand factor (via a D'-D3 fragment), and benefiting from the pharmacokinetic prolongation provided by the addition of hydrophilic polypeptides, efanesoctocog alfa opens a new era in the treatment of hemophilia A.The term Ultra-Long (UL) FVIII has been proposed to designate it and differentiate it from EHL FVIII. The level of FVIII correction within the normal range for several days provided by this molecule should allow an increasing number of patients to mentally and physically free themselves from the physical and psychological constraints of hemophilia A. Certainly, the constraint of weekly intravenous infusions persists, but is compensated by a correction of hemostasis whose amplitude and duration remain unmatched by other therapeutic options currently available.