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
Agenda of the PRAC meeting 8-11 April 2024
Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive
Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide} Treatment of Netherton syndrome, 17/10/2019 Positive
Orphan designation: Nirogacestat Treatment of soft tissue sarcoma, 17/10/2019 Positive
Orphan designation: Adeno-associated virus serotype rh10 containing the human GALC gene Treatment of Krabbe disease, 15/10/2021 Positive
Orphan designation: Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Treatment of Duchenne muscular dystrophy, 14/10/2016 Positive
Orphan designation: Olorofim Treatment of invasive Scopulariopsis, 14/01/2022 Positive