Human medicines European public assessment report (EPAR): Vargatef, nintedanib, Date of authorisation: 21/11/2014, Revision: 20, Status: Authorised

Orphan designation: Autologous induced pluripotent stem cells-derived thymic epithelial cells transduced with a lentiviral vector encoding forkhead box protein N1 Treatment of DiGeorge syndrome, 24/05/2024 Positive

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene Treatment of recombination-activating gene 1 (RAG1) deficiency, 24/05/2024 Positive

Orphan designation: N-(1,3-Benzothiazol-2-yl)-4-[(2-hydroxy-3-methoxyphenyl)methylamino]benzenesulfonamide Treatment of Platelet-activating anti-Platelet factor 4 (PF4) disorders, 24/05/2024 Positive

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