Orphan designation: Florilglutamic acid (18F) Diagnosis of hepatocellular carcinoma, 21/03/2016 Withdrawn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homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn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CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn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F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn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
Orphan designation: Allogeneic cultured postnatal thymus-derived tissue Treatment of CHARGE syndrome, 26/02/2019 Positive
Orphan designation: Azithromycin dihydrate Prevention of bronchopulmonary dysplasia, 14/01/2022 Positive
Orphan designation: Mavorixafor Treatment of WHIM syndrome, 25/07/2019 Positive