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

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

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

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

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

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

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

Orphan designation: Adeno-associated virus vector serotype 8 containing the human TYMP gene (CpG-depleted) Treatment of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE), 25/07/2024 Positive

Orphan designation: (E)-2-((4-((4-Benzyl(ethyl)amino)phenyl)diazinyl)phenyl)amino-N,N,N-triethyl-2-oxoethan-1-aminium chloride Treatment of non-syndromic inherited retinal dystrophies of the rod-dominant phenotype, 25/07/2024 Positive

Orphan designation: Rivoceranib (mesilate) Treatment of hepatocellular carcinoma, 25/07/2024 Positive