Orphan designation: Ganaxolone Treatment of CDKL5 deficiency disorder, 13/11/2019 Positive
Orphan designation: single guide RNA targeting the human TTR gene, Ziclumeran Treatment of ATTR amyloidosis, 26/03/2021 Positive
Orphan designation: Ganaxolone Treatment of tuberous sclerosis, 15/10/2021 Positive
Orphan designation: Resminostat Treatment of Hodgkin's lymphoma, 09/12/2011 Withdrawn
Orphan designation: recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene Treatment of Leber's congenital amaurosis, 16/10/2017 Withdrawn
Orphan designation: Resminostat Treatment of hepatocellular carcinoma, 27/09/2011 Withdrawn
Orphan designation: human decorin fused to the truncated homing peptide CRK Treatment of epidermolysis bullosa, 18/07/2022 Positive
Orphan designation: posnafusp alfa Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome), 14/01/2022 Positive
Orphan designation: autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human claudin 18.2 antigen with CD28 and CD3-zeta intracellular signalling domains Treatment of gastric cancer, 19/02/2021 Positive
Orphan designation: small-interfering RNA against soluble fms-like tyrosine kinase-1 e15a mRNA, small-interfering RNA against soluble fms-like tyrosine kinase-1 i13 mRNA Treatment of pre-eclampsia, 16/04/2025 Positive