Orphan designation: Trehalose Treatment of neuronal ceroid lipofuscinosis, 19/10/2020 Withdrawn
Orphan designation: Humanised IgG4 monoclonal antibody against C1q Treatment of Guillain-Barré syndrome, 10/02/2025 Positive
Orphan designation: sutimlimab Treatment of autoimmune haemolytic anaemia, 11/02/2025 Positive
Orphan designation: Upifitamab rilsodotin Treatment of ovarian cancer, 09/12/2022 Withdrawn
Orphan designation: pelabresib monohydrate Treatment of myelofibrosis, 17/02/2025 Positive
Orphan designation: Adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene Treatment of Huntington’s disease, 01/04/2019 Withdrawn
Orphan designation: Ornithine phenylacetate Treatment of acute liver failure, 20/02/2025 Positive
Orphan designation: Serplulimab Treatment of small cell lung cancer, 20/02/2025 Positive
Orphan designation: autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 Treatment of myasthenia gravis, 20/02/2025 Positive
Orphan designation: Seladelpar Treatment of primary biliary cholangitis, 20/02/2025 Positive