Orphan designation: Mesenchymal stem cells-derived small extracellular vesicles loaded with siRNA against phosphatase and tensin homolog Treatment of spinal cord injury, 11/11/2024 Positive

Orphan designation: Autologous bone marrow derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene Treatment of X-linked severe combined immunodeficiency, 13/11/2020 Withdrawn

Orphan designation: Autologous mobilised peripheral blood-derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene Treatment of X-linked severe combined immunodeficiency, 20/05/2021 Withdrawn

Orphan designation: Treatment of Becker muscular dystrophy, 26/06/2020 Withdrawn

Orphan designation: 6-(4-(tert-butyl)phenoxy)pyridin-3-amine Treatment of acute lymphoblastic leukaemia, 10/12/2021 Withdrawn

Orphan designation: Marstacimab Treatment of haemophilia B, 13/12/2023 Withdrawn

Orphan designation: H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH Treatment of haemophilia A, 10/08/2022 Withdrawn

Orphan designation: glecirasib Treatment of pancreatic cancer, 10/11/2024 Positive

Orphan designation: octreotide hydrochloride Treatment of autosomal dominant polycystic liver disease, 10/11/2024 Positive

Nitrosamine impurities in specific medicines