Orphan designation: apilimod Treatment of amyotrophic lateral sclerosis, 28/06/2024 Positive

Orphan designation: Ethyl(2E,4S)-4-({(2S)-2-[3-{[(5-methyl-1,2-oxazol-3-yl)carbonyl]amino}-2-oxopyridin-1(2H)-yl]pent-4-ynoyl}amino)-5-[(3S)-2-oxopyrrolidin-3-yl]pent-2-enoate, Pocapavir Treatment of poliovirus infection, 28/06/2024 Positive

Orphan designation: (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone Treatment of pachyonychia congenita, 28/06/2024 Positive

Human medicines European public assessment report (EPAR): Vaxzevria (previously COVID-19 Vaccine AstraZeneca), COVID-19 Vaccine (ChAdOx1-S [recombinant]), Date of authorisation: 29/01/2021, Revision: 32, Status: Withdrawn (authorisation)

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