Orphan designation: brogidirsen Treatment of Duchenne muscular dystrophy, 13/12/2023 Positive

Orphan designation: Humanised IgG1 kappa fragment antibody targeting TfR1 conjugated to P125 oligonucleotide Treatment of myotonic disorders, 22/05/2023 Positive

Orphan designation: Govorestat Treatment of Charcot-Marie-Tooth disease, 22/05/2023 Positive

Orphan designation: 2-((2-fluoro-4-iodophenyl)amino)-N-(2-hydroxyethoxy)-1-methyl-1H-pyrrolo[2,3-b]pyridine-3-carboxamide Treatment of neurofibromatosis type 1, 22/05/2023 Positive

Orphan designation: Fingolimod Treatment of hypomyelinating leukodystrophy-18, 22/05/2023 Positive

Orphan designation: Efzofitimod Treatment of systemic sclerosis, 20/06/2023 Positive

Orphan designation: Fasudil hydrochloride Treatment of amyotrophic lateral sclerosis, 20/06/2023 Positive

Orphan designation: Adeno-associated virus vector serotype 8 encoding the ABCA4 protein, C-region, adeno-associated virus vector serotype 8 encoding the ABCA4 protein, N-region Treatment of inherited retinal dystrophies, 20/06/2023 Positive

Orphan designation: (3S,3'S,3a'S,10a'S)-6-chloro-3'-(3-chloro-2-fluorophenyl)-1'-(cyclopropylmethyl)-6'-methyl-2-oxo-1,2,3',3a',10',10a'-hexahydro-1'H-spiro[indole-3,2'-pyrrolo[2',3':4,5]pyrrolo[1,2-b]indazole]-7'-carboxylic acid Treatment of soft tissue sarcoma, 20/06/2023 Positive

Orphan designation: Evorpacept Treatment of gastric cancer, 20/06/2023 Positive