Orphan designation: lentiviral vector containing the human MMUT transgene Treatment of methylmalonic acidaemia, 22/08/2025 Positive

Orphan designation: 2'-O-(2-Methoxyethyl) modified antisense oligonucleotide against MECP2 pre-mRNA Treatment of MECP2 duplication syndrome, 21/11/2025 Positive

Orphan designation: cladribine Treatment of acute myeloid leukaemia, 22/08/2025 Positive

Orphan designation: leniolisib Treatment of common variable immunodeficiency (CVID), 22/08/2025 Positive

Orphan designation: 2-(3-Chlorophenyl)-N-[6-(trifluoromethyl)-1,3-benzothiazol-2-yl]acetamide Treatment of amyotrophic lateral sclerosis, 21/11/2025 Positive

Orphan designation: 3-tert-butyl-N-{(1R)-1-[4-(6-{6-[4-({1-[4-(2,4-dioxo-1,3-diazinan-1-yl)phenyl]piperidin-4-yl}methyl)piperazin-1-yl]pyridin-3-yl}-7H-pyrrolo[2,3-d]pyrimidin-4-yl)-2-methylphenyl]ethyl}-1,2,4-oxadiazole-5-carboxamide Treatment of lymphoplasmacytic lymphoma, 22/08/2025 Positive

Orphan designation: sonpiretigene isteparvovec Treatment of non-syndromic macular dystrophy, 22/08/2025 Positive

Orphan designation: brepocitinib Treatment of idiopathic inflammatory myopathy, 22/08/2025 Positive

Orphan designation: antisense oligonucleotide against ABCA4 pre-mRNA Treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene, 21/11/2025 Positive

Orphan designation: zanidatamab Treatment of oesophageal cancer, 21/11/2025 Positive