Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Orphan designation: Adeno-associated viral vector containing modified U1 snRNA Treatment of Duchenne muscular dystrophy, 08/10/2009 Positive

Orphan designation: Florilglutamic acid (18F) Diagnosis of glioma, 21/03/2016 Withdrawn

Orphan designation: Florilglutamic acid (18F) Diagnosis of hepatocellular carcinoma, 21/03/2016 Withdrawn

Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Orphan designation: 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F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Minutes of the PRAC meeting 5-8 February 2024

Orphan designation: Allogeneic cultured postnatal thymus-derived tissue Treatment of DiGeorge syndrome, 26/02/2019 Positive