Giroctocogene fitelparvovec for haemophilia A

Giroctocogene fitelparvovec is in development for the treatment of moderately severe to severe
haemophilia A in adult males. Haemophilia A is a rare bleeding disorder, caused by lack of blood
clotting factor VIII which facilitates blood clotting and reduces bleeding events. The absence of
factor VIII makes patients prone to excessive and uncontrolled bleeding. In severe cases, this can
result in spontaneous bleeding into the joints, muscles or brain causing serious complications. The
inability of their blood to clot means that patients are at high risk of internal bleeding, including
from the brain, increasing their mortality risk. Haemophilia A is treated with replacement factor
VIII (FVIII) to restore blood clotting and prevent bleeding. Most of the current treatments include
frequent intravenous infusions with FVIII concentrate for disease prevention which can affect
patient adherence. Therefore, there is a need to develop additional treatment options to increase
adherence in these patients.