valoctocogene roxaparvovec + corticosteroids

This drug is a **combination therapy** of valoctocogene roxaparvovec, a gene therapy, with corticosteroids. - **Valoctocogene roxaparvovec** is an adeno-associated virus (AAV) serotype 5 vector-based gene therapy delivering a B-domain deleted human factor VIII (hFVIII-SQ) transgene under a liver-specific promoter, restoring endogenous production of factor VIII for patients with severe hemophilia A (congenital factor VIII deficiency)[1][5][6]. The transgene persists episomally in hepatocytes and increases circulating factor VIII for up to 5 years[1][5]. - **Corticosteroids** are co-administered prophylactically or reactively to mitigate the immune-mediated elevation of liver enzymes (transaminitis) that often occurs after AAV gene therapy[1][2][4][5]. Their use helps preserve factor VIII activity by suppressing hepatic immune responses[2][4]. The regimen is typically intravenous gene therapy infusion (valoctocogene roxaparvovec) and oral corticosteroids started if alanine transaminase (ALT) rises after treatment[1][4][5]. Combined, this therapy is used for adults with severe hemophilia A, aiming to achieve sustained endogenous factor VIII expression while minimizing hepatic inflammation and decline in factor VIII activity levels[1][2][4][5].

Other names

valoctocogene roxaparvovec-rvoxvaloctogene roxaparvovec + corticosteroids