Active substance Disease/condition EU designation number
Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A EU/3/18/2015
Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene Treatment of haemophilia A EU/3/16/1622
Recombinant adeno-associated viral vector containing a bioengineered capsid and a codon-optimised expression cassette to drive the expression of the SQ form of a B-domain deleted human coagulation factor VIII Treatment of haemophilia A EU/3/18/2079
Recombinant adeno-associated viral vector serotype 6 encoding the B-domain-deleted human factor VIII Treatment of haemophilia A EU/3/17/1874
Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B- Treatment of haemophilia B EU/3/15/1599
Fidanacogene elaparvovec Treatment of haemophilia B EU/3/18/2090
Lentiviral vector encoding human coagulation factor IX Treatment of haemophilia B EU/3/19/2141
Adeno-associated viral vector containing the human factor-IX gene Treatment of haemophilia B EU/3/11/938
Recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA Treatment of haemophilia B EU/3/18/1999
Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B EU/3/18/2080