Transitioning from emicizumab prophylaxis to valoctocogene roxaparvovec gene therapy: A simulation study for individuals with severe haemophilia A.

Suresh AgarwalCedric HermansWolfgang A Miesbach Flora Peyvandi Robert F SidonioDane OsmondVanessa NewmanJosh HenshawSteven W Pipe

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2024)

Pharmacokinetic simulations demonstrated no clinically meaningful difference in bleeding risk caused by decaying emicizumab levels and rising gene therapy-derived endogenous FVIII for all examined emicizumab doses and dosing regimens. Therefore, multiple approaches can safely transition individuals from emicizumab prophylaxis to valoctocogene roxaparvovec.

Keyphrases

gene therapy
molecular dynamics
early onset
drug induced