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

Orphan designation: camrelizumab Treatment of hepatocellular carcinoma, 25/07/2024 Positive

Orphan designation: Heterologous swine glyco-humanised polyclonal antibody against T lymphocytes Treatment of peripheral T-cell lymphoma, 25/07/2024 Positive

Human medicines European public assessment report (EPAR): Tauvid, Flortaucipir (18F), Date of authorisation: 22/08/2024, Status: Authorised