For help on how to get the results you want, see our search tips.
568 results
Medicine
European public assessment reports (EPAR) Remove European public assessment reports (EPAR) filter
Orphan designations Remove Orphan designations filter
Categories
Human Remove Human filter
Orphan designation status
Withdrawn Remove Withdrawn filter
-
List item
Orphan designation: 5-(Ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (ezutromid) for: Treatment of Duchenne muscular dystrophy (updated)
Date of first decision: 04/12/2008, Withdrawn, Last updated: 10/02/2021 -
List item
Orphan designation: Human monoclonal antibody against Pseudomonas aeruginosa IATS-O1 for: Treatment of pneumonia caused by serotype O1 Pseudomonas aeruginosa (updated)
Date of first decision: 28/01/2010, Withdrawn, Last updated: 03/02/2021 -
List item
Orphan designation: RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3'-[2'a-[N2-acetyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-6-aminohexanoyl-β-alanyl], octahydrochloride for: Treatment of Duchenne muscular dystrophy (updated)
Date of first decision: 02/02/2010, Withdrawn, Last updated: 03/02/2021 -
List item
Orphan designation: Sodium thiosulfate for: Treatment of calciphylaxis (updated)
Date of first decision: 23/02/2011, Withdrawn, Last updated: 03/02/2021 -
List item
Orphan designation: Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec) for: Treatment of Crigler-Najjar syndrome (updated)
Date of first decision: 18/11/2016, Withdrawn, Last updated: 03/02/2021 -
List item
Orphan designation: Heparin-activated recombinant human fibroblast growth factor 1 (on a biodegradable device made from alpha-calcium sulfate hemihydrate) for: Treatment of traumatic spinal cord injury (updated)
Date of first decision: 27/07/2010, Withdrawn, Last updated: 03/02/2021 -
List item
Orphan designation: (1S,3S)-3-amino-4-(difluoromethylene) cyclopentanecarboxylic acid hydrochloride for: Treatment of West syndrome (updated)
Date of first decision: 09/02/2012, Withdrawn, Last updated: 03/02/2021 -
List item
Orphan designation: Rovalpituzumab tesirine for: Treatment of small cell lung cancer (updated)
Date of first decision: 30/05/2016, Withdrawn, Last updated: 03/02/2021 -
List item
Orphan designation: rasagiline for: Treatment of Duchenne muscular dystrophy
Date of first decision: 28/06/2019, Withdrawn, Last updated: 12/01/2021 -
List item
Orphan designation: Tetrahydrobiopterin for: Treatment of hyperphenylalaninaemia
Date of first decision: 08/06/2004, Withdrawn, Last updated: 11/01/2021 -
List item
Orphan designation: adeno-associated viral vector serotype 2 containing the human REP1 gene (timrepigene emparvovec) for: Treatment of spinal muscular atrophy
Date of first decision: 24/08/2018, Withdrawn, Last updated: 07/01/2021 -
List item
Orphan designation: Antisense oligonucleotide targeting the F508delta mutation of CFTR (eluforsen) for: Treatment of cystic fibrosis
Date of first decision: 07/10/2013, Withdrawn, Last updated: 18/12/2020 -
List item
Orphan designation: 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor) for: Treatment of systemic sclerosis
Date of first decision: 19/11/2014, Withdrawn, Last updated: 14/12/2020 -
List item
Orphan designation: 5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate] for: Treatment of biliary tract cancer
Date of first decision: 16/10/2017, Withdrawn, Last updated: 11/12/2020 -
List item
Orphan designation: Recombinant antibody derivative against human CD19 and CD3 (blinatumomab) for: Treatment of chronic lymphocytic leukaemia
Date of first decision: 01/12/2003, Withdrawn, Last updated: 11/12/2020 -
List item
Orphan designation: Recombinant antibody derivative against human CD19 and CD3 (blinatumomab) for: Treatment of mantle cell lymphoma
Date of first decision: 01/12/2003, Withdrawn, Last updated: 11/12/2020 -
List item
Orphan designation: pentetrazol for: Treatment of idiopathic hypersomnia
Date of first decision: 11/11/2015, Withdrawn, Last updated: 10/12/2020 -
List item
Orphan designation: copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin for: Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy
Date of first decision: 27/06/2013, Withdrawn, Last updated: 10/12/2020 -
List item
Orphan designation: Ecothiopate iodide for: Treatment of Stargardt's disease
Date of first decision: 24/04/2015, Withdrawn, Last updated: 10/12/2020 -
List item
Orphan designation: Allantoin for: Treatment of epidermolysis bullosa
Date of first decision: 16/01/2014, Withdrawn, Last updated: 10/12/2020 -
List item
Orphan designation: Alpha-1 proteinase inhibitor for: Treatment of cystic fibrosis
Date of first decision: 14/09/2007, Withdrawn, Last updated: 08/12/2020 -
List item
Orphan designation: Sodium cromoglicate for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 26/06/2020, Withdrawn, Last updated: 08/12/2020 -
List item
Orphan designation: L-cysteine, L-leucyl-L-alpha-glutamyl-L-alpha-glutamyl-L-lysyl-L-lysylglycyl-L-asparaginyl-L-tyrosyl-L-valyl-L-valyl-L-threonyl-L-alpha-aspartyl-L-histidyl-S-[1-[(4-carboxycyclohexyl)methyl]-2,5-dioxo-3-pyrrolidinyl]-complex with keyhole limpet haemocyanin for: Treatment of glioma
Date of first decision: 27/10/2011, Withdrawn, Last updated: 04/12/2020 -
List item
Orphan designation: Adeno-associated viral vector serotype Anc80 containing the truncated human ATP7B gene under the control of the human alpha-1 antitrypsin promoter for: Treatment of Wilson's disease
Date of first decision: 23/08/2017, Withdrawn, Last updated: 03/12/2020 -
List item
Orphan designation: Sodium (2R,3S,5R)-5-(4-amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5-(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetrahydrofuran-2-yl)methyl phosphate (guadecitabine) for: Treatment of acute myeloid leukaemia
Date of first decision: 14/12/2015, Withdrawn, Last updated: 01/12/2020