Orphan designation: 5-bromo-N-(prop-2-yn-1-yl)-2-(1H-1,2,4-triazol-1-yl)pyrimidine-4,6-diamine Treatment of Prader-Willi syndrome, 08/11/2023 Positive

Orphan designation: Ambroxol hydrochloride Treatment of Gaucher disease, 08/11/2023 Positive

Orphan designation: bortezomib Treatment of patients with light chain (AL) amyloidosis, 08/11/2023 Positive

Orphan designation: setanaxib Treatment of Alport syndrome, 08/11/2023 Positive

Orphan designation: Methyl 4-(2-acetamidoethylsulfanyl)-4-oxobutanoate Treatment of Leigh syndrome, 13/12/2023 Positive

Orphan designation: Allogeneic amniotic fluid-derived mesenchymal stem cells with lung specificity Prevention of primary graft dysfunction following lung transplantation, 13/12/2023 Positive

Orphan designation: golcadomide hydrochloride Treatment of follicular lymphoma, 13/12/2023 Positive

Orphan designation: Selumetinib Treatment of glioma, 13/12/2023 Positive

Orphan designation: cutamesine Treatment of amyotrophic lateral sclerosis, 13/12/2023 Positive

Orphan designation: Ovine polyclonal fragment antigen-binding against ricin Treatment of ricin poisoning, 13/12/2023 Positive