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

Agenda - Executive Steering Group on Shortages and Safety of Medicinal Products (MSSG) (18 September 2024)

Orphan designation: Cannabidiol acid methyl ester Treatment of Prader-Willi syndrome, 25/07/2024 Positive

EMEA-003316-PIP01-22

Human medicines European public assessment report (EPAR): Adenuric, febuxostat, Date of authorisation: 21/04/2008, Revision: 23, Status: Authorised

EMEA-003346-PIP01-22