Orphan designation: N-[(1R)-1-[(S)-(2-Chloro-3-fluorophenyl)hydroxymethyl]butyl]-7-fluoro-2,3-dihydro-2-oxo-1H-indole-4-carboxamide Treatment of congenital alpha-1 antitrypsin deficiency, 25/07/2024 Positive

13 September 2024

Orphan designation: N,N'-([Cyclohexylmethylene]di-4,1-phenylene)bis(2-[1-pyrrolidinyl]acetamide) Treatment of Creutzfeldt-Jakob disease, 25/07/2024 Positive

13 September 2024

Orphan designation: emavusertib Treatment of primary large B-cell lymphoma of immune-privileged sites, 25/07/2024 Positive

13 September 2024

Orphan designation: Sodium phenylacetate,Ursodoxicoltaurine Treatment of Wolfram syndrome, 25/07/2024 Positive

13 September 2024

Orphan designation: Adeno-associated virus vector serotype rh.10 containing the human FXN gene Treatment of Friedreich's ataxia, 25/07/2024 Positive

13 September 2024

Orphan designation: 4-[[[4-[5-Chloro-2-[[trans-4-[[(1R)-2-methoxy-1-methyl-ethyl]amino]cyclohexyl]amino]-4-pyridinyl]-2-thiazolyl]amino]methyl]tetrahydro-2H-pyran-4-carbonitrile Treatment of peripheral T-cell lymphoma, 25/07/2024 Positive

13 September 2024

Orphan designation: befiradol fumarate Treatment of spinocerebellar ataxia, 25/07/2024 Positive

13 September 2024

Orphan designation: Adeno-associated virus serotype A101 containing the codon-optimized human CFTRdeltaR Treatment of cystic fibrosis, 25/07/2024 Positive

13 September 2024

Orphan designation: afamelanotide Treatment of variegate porphyria, 25/07/2024 Positive

13 September 2024

Orphan designation: Urokinase, catalytic domain, fused with a single-chain antibody against von Willebrand factor Treatment of thrombotic thrombocytopenic purpura, 25/07/2024 Positive

13 September 2024