Orphan designation: admilparant Treatment of idiopathic pulmonary fibrosis, 22/08/2025 Positive

Orphan designation: 4-(2-{[2-Amino-6-(3-chloro-2-methylphenyl)pyrimidin-4-yl]amino}ethyl)benzene-1-sulfonamide Treatment of acute myeloid leukaemia, 22/08/2025 Positive

Orphan designation: allogeneic cultured postnatal thymus-derived tissue Treatment of congenital athymia, 22/08/2025 Positive

Orphan designation: gildeuretinol, gildeuretinol acetate Treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene, 22/08/2025 Positive

Orphan designation: RNA editing antisense oligonucleotide against the Z mutation of the human SERPINA1 mRNA transcript, sodium salt Treatment of alpha-1 antitrypsin deficiency, 22/08/2025 Positive

Orphan designation: adeno-associated viral vector serotype ShH10 containing a codon-optimised human NDP gene Treatment of NDP gene-related disorders Positive

Orphan designation: IgG1 trispecific monoclonal antibody against T-cell receptor CD3, B-cell maturation antigen and G protein-coupled receptor class C group 5 member D Treatment of multiple myeloma Positive

Orphan designation: sonpiretigene isteparvovec Treatment of syndromic inherited retinal dystrophies of the rod-dominant phenotype, 22/08/2025 Positive

Orphan designation: darovasertib Treatment of uveal melanoma, 22/08/2025 Positive

Orphan designation: acetylcysteine amide Treatment of hereditary cerebral amyloid angiopathies, 22/08/2025 Positive