Orphan designation: Veliparib Treatment of ovarian cancer, 17/12/2010 Withdrawn

Orphan designation: Eflornithine Treatment of neuroblastoma, 27/09/2011 Withdrawn

Orphan designation: Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor for the treatment of ovarian cancer Treatment of ovarian cancer, 29/04/2014 Withdrawn

Orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn

Orphan designation: Adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene Treatment of aspartylglucosaminuria, 19/10/2020 Withdrawn

Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- Treatment of cystic fibrosis, 14/10/2016 Withdrawn

Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn

Orphan designation: 3-(((1S,2S,3R)-2,3-difluoro-1-hydroxy-7-(methylsulfonyl)-2,3-dihydro-1H-inden-4-yl)oxy)-5-fluorobenzonitrile Treatment of von Hippel-Lindau disease, 21/08/2020 Withdrawn

Orphan designation: Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane Treatment of idiopathic pulmonary fibrosis, 21/08/2020 Withdrawn

Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn