Orphan designation: Complement factor H Treatment of C3 glomerulopathy, 28/06/2024 Positive
Orphan designation: Complement factor H Treatment of C3 glomerulopathy, 28/06/2024 Positive
Orphan designation: Complement factor H Treatment of C3 glomerulopathy, 28/06/2024 Positive
Orphan designation: L-methionine Treatment of pulmonary alveolar proteinosis, 28/06/2024 Positive
Orphan designation: sargramostim Treatment of pulmonary alveolar proteinosis, 28/06/2024 Positive
Orphan designation: (R)-3-(1-Cyclopropyl-3-(2-fluoro-4-(trifluoromethoxy)benzyl)ureido)piperidine-1-carboxamide Treatment of hyperphenylalaninaemia, 28/06/2024 Positive
Orphan designation: apilimod Treatment of amyotrophic lateral sclerosis, 28/06/2024 Positive
Orphan designation: Ethyl(2E,4S)-4-({(2S)-2-[3-{[(5-methyl-1,2-oxazol-3-yl)carbonyl]amino}-2-oxopyridin-1(2H)-yl]pent-4-ynoyl}amino)-5-[(3S)-2-oxopyrrolidin-3-yl]pent-2-enoate, Pocapavir Treatment of poliovirus infection, 28/06/2024 Positive
Orphan designation: (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone Treatment of pachyonychia congenita, 28/06/2024 Positive
Human medicines European public assessment report (EPAR): Vaxzevria (previously COVID-19 Vaccine AstraZeneca), COVID-19 Vaccine (ChAdOx1-S [recombinant]), Date of authorisation: 29/01/2021, Revision: 32, Status: Withdrawn (authorisation)
Human medicines European public assessment report (EPAR): Vargatef, nintedanib, Date of authorisation: 21/11/2014, Revision: 20, Status: Authorised
Orphan designation: Autologous induced pluripotent stem cells-derived thymic epithelial cells transduced with a lentiviral vector encoding forkhead box protein N1 Treatment of DiGeorge syndrome, 24/05/2024 Positive