If approved, valoctocogene roxaparvovec would be the first gene therapy in the US for this indication.
People living with haemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk of painful and potentially life-threatening bleeds from even modest injuries.
Patients with the most severe form of the disease, which make up approximately 50% of the haemophilia A population, are currently treated with a prophylactic regimen of intravenous Factor VIII infusions administered two to three times per week or a bispecific monoclonal antibody that mimics the activity of Factor VIII, which is administered one to four times per month.
