Orphan designation: Adeno-associated virus serotype rh.74 vector containing the human PKP2 gene isoform Treatment of arrhythmogenic cardiomyopathy caused by pathogenic mutations in the PKP2 gene, 24/05/2024 Positive

Orphan designation: afamelanotide Treatment of xeroderma pigmentosum, 24/05/2024 Positive

Orphan designation: 2,4-Diamino-5-[[5-(1H-pyrazol-5-yl)-2-thienyl]methyl]-1H-pyrimidin-6-one Treatment of hyperphenylalaninaemia, 24/05/2024 Positive

Orphan designation: DNA plasmid containing the COL7A1 gene Treatment of epidermolysis bullosa, 24/05/2024 Positive

Orphan designation: serdexmethylphenidate Treatment of idiopathic hypersomnia, 24/05/2024 Positive

Orphan designation: efineptakin alfa Treatment of acute radiation syndrome, 24/05/2024 Positive

Orphan designation: Adeno-associated virus serotype 9 containing CRISPR/Cas13Y and guide RNA against the human MECP2 gene Treatment of MECP2 duplication syndrome, 24/05/2024 Positive

Human medicines European public assessment report (EPAR): GalliaPharm, Gallium (68Ga) chloride,Germanium (68Ge) chloride, Date of authorisation: 01/08/2024, Status: Authorised

Minutes of the COMP meeting 18-20 June 2024

Human medicines European public assessment report (EPAR): Tremelimumab AstraZeneca, tremelimumab, Date of authorisation: 20/02/2023, Revision: 1, Status: Withdrawn (authorisation)