Orphan designation: 7-Ethyl-10-hydroxycamptothecin, Irinotecan hydrochloride trihydrate Treatment of small cell lung cancer, 24/05/2024 Positive
Orphan designation: Allogeneic non-alloreactive T cells edited with mRNA to disrupt TRAC and CD52 genes and transduced with lentiviral vector expressing a chimeric antigen receptor against CD22 and RQR8 depletion mechanism Treatment of acute lymphoblastic leukaemia, 24/05/2024 Positive
Orphan designation: quabodepistat Treatment of tuberculosis, 24/05/2024 Positive
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