Orphan designation: nangibotide Treatment of sickle cell disease, 21/11/2025 Positive
Orphan designation: Felzartamab Treatment of primary membranous nephropathy, 21/11/2025 Positive
Orphan designation: Infigratinib Treatment of hypochondroplasia, 21/11/2025 Positive
Orphan designation: resecabtagene autoleucel Treatment of systemic sclerosis, 21/11/2025 Positive
Orphan designation: soquelitinib Treatment of peripheral T-cell lymphoma, 20/10/2025 Positive
Orphan designation: bevantolol hydrochloride Treatment of Huntington's disease, 20/10/2025 Positive
Orphan designation: orziloben Treatment of intestinal failure-associated liver disease Positive
Orphan designation: aviptadil Treatment of acute respiratory distress syndrome (ARDS), 23/02/2026 Positive
Creon
Orphan designation: 1-{[2-(3,6-Diazabicyclo[3.1.1]heptan-3-yl)-7-(1,3-thiazol-2-yl)-1,3-benzoxazol-4-yl]oxy}-1,1-difluoro-2-methylpropan-2-ol Treatment of palmoplantar pustulosis Positive