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

Orphan designation: emugrobart Treatment of spinal muscular atrophy, 22/08/2025 Positive

Orphan designation: RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a->5') (C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-[2'a-[39-[[1-acetyl-L-prolyl-L-lysyl-L-lysyl-L-lysyl-L-arginyl-L-lysyl-L-valyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-(L-phenylalanylglycyl-L-phenylalanylglycyl-L-arginylglycyl-L-arginyl-L-gamma-glutamyl)-L-lysyl]amino]-1- oxo-4,7,10,13,16,19,22,25,28,31,34,37-dodecaoxanonatriacont-1-yl]]G), (8'->1')-lactam Treatment of dystrophic myotonia, 22/08/2025 Positive

Orphan designation: rilzabrutinib Treatment of immunoglobulin G4-related disease, 22/08/2025 Positive

Orphan designation: adeno-associated virus serotype 9 vector containing the human NDP gene Treatment of NDP gene-related disorders, 22/08/2025 Positive

Orphan designation: mezagitamab Treatment of immune thrombocytopenia, 22/08/2025 Positive

Orphan designation: 7-ethyl-10-hydroxycamptothecin Treatment of pancreatic cancer, 22/08/2025 Positive

Orphan designation: pegtarazimod Treatment of graft-versus-host disease, 22/08/2025 Positive