Orphan designation: Adeno-associated virus serotype 5 vector encoding C1-esterase inhibitor Treatment of hereditary angioedema, 10/11/2022 Withdrawn

Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel) Treatment of Wiskott-Aldrich syndrome, 07/10/2013 Withdrawn

Orphan designation: amitriptyline Treatment of erythromelalgia, 21/06/2022 Withdrawn

Orphan designation: Adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene Treatment of mucopolysaccharidosis type II (Hunter's syndrome), 10/08/2015 Withdrawn

Orphan designation: Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase-alpha gene Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome), 24/01/2013 Withdrawn

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency, 26/03/2014 Positive

Appendix 1: Acceptable intakes established for N-nitrosamines

Orphan designation: zopapogene imadenovec Treatment of recurrent respiratory papillomatosis, 12/01/2024 Positive

Orphan designation: N-(2-Methoxyethyl)-6-methyl-N-[(3-methyl-2-thienyl)methyl]-2-oxo-1,2-dihydropyridine-4-carboxamide Treatment of glycogen storage disease type IV, 16/01/2025 Positive

Orphan designation: volixibat potassium Treatment of primary biliary cholangitis, 16/01/2025 Positive