For help on how to get the results you want, see our search tips.
1713 results
Medicine
European public assessment reports (EPAR) Remove European public assessment reports (EPAR) filter
Summaries of opinion Remove Summaries of opinion filter
Orphan designations Remove Orphan designations filter
Categories
Human Remove Human filter
Orphan designation status
Positive Remove Positive filter
-
List item
Orphan designation: Human monoclonal antibody targeting Staphylococcus aureus alpha-toxin (tosatoxumab) for: Treatment of pneumonia caused by Staphylococcus aureus
Date of first decision: 05/03/2012, Positive, Last updated: 27/08/2020 -
List item
Orphan designation: Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol for: Treatment of cystic fibrosis
Date of first decision: 30/08/2011, Positive, Last updated: 27/08/2020 -
List item
Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against p53 mRNA (teprasiran) for: Prevention of delayed graft function after renal transplantation
Date of first decision: 06/06/2010, Positive, Last updated: 27/08/2020 -
List item
Orphan designation: Human monoclonal antibody against Pseudomonas aeruginosa serotype O11 (Panobacumab) for: Treatment of pneumonia caused by serotype O11 Pseudomonas aeruginosa
Date of first decision: 29/06/2006, Positive, Last updated: 27/08/2020 -
List item
Orphan designation: Pevonedistat for: Treatment of acute myeloid leukaemia
Date of first decision: 25/07/2019, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: Pevonedistat for: Treatment of myelodysplastic syndromes
Date of first decision: 14/12/2018, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E (brentuximab vedotin) for: Adjunctive treatment in haematopoietic cell transplantation
Date of first decision: 15/01/2009, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E for: Treatment of peripheral T-cell lymphoma
Date of first decision: 21/09/2019, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: Ixazomib for: Treatment of systemic light chain amyloidosis
Date of first decision: 08/11/2012, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue (darvadstrocel) for: Treatment of anal fistula
Date of first decision: 08/10/2009, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: Brentuximab vedotin for: Treatment of cutaneous T-cell lymphoma
Date of first decision: 11/01/2012, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: Balipodect for: Adjunctive treatment in haematopoietic cell transplantation
Date of first decision: 14/04/2019, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: 5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride for: Treatment of Kabuki syndrome
Date of first decision: 19/11/2018, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: 6-{[(1R,2S)-2-aminocyclohexyl]amino}-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridin-3-one monocitrate for: Treatment of acute myeloid leukaemia
Date of first decision: 22/02/2018, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: 2,2'-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid for: Treatment of multiple myeloma
Date of first decision: 27/09/2011, Positive, Last updated: 26/08/2020 -
List item
Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate for: Treatment of mucopolysaccharidosis type I
Date of first decision: 22/09/2016, Positive, Last updated: 25/08/2020 -
List item
Orphan designation: "Synthetic 47-amino-acid N-myristoylated lipopeptide, derived from the preS region of hepatitis B virus (bulevirtide) for: Treatment of hepatitis delta virus infection
Date of first decision: 19/06/2015, Positive, Last updated: 12/08/2020 -
List item
Orphan designation: Humanised monoclonal antibody to the folate receptor alpha for: Treatment of ovarian cancer
Date of first decision: 01/04/2008, Positive, Last updated: 11/08/2020 -
List item
Orphan designation: Amatuximab for: Treatment of malignant mesothelioma
Date of first decision: 16/01/2014, Positive, Last updated: 11/08/2020 -
List item
Orphan designation: N-{2-[(6-{[(2,6-dichloro-3,5-dimethoxyphenyl)carbamoyl](methyl)amino}pyrimidin-4-yl)amino]-5-(4-ethylpiperazin-1-yl)phenyl}prop-2-enamide for: Treatment of hepatocellular carcinoma
Date of first decision: 23/08/2017, Positive, Last updated: 11/08/2020 -
List item
Orphan designation: Denileukin diftitox for: Treatment of cutaneous T-cell lymphoma
Date of first decision: 11/12/2001, Positive, Last updated: 11/08/2020 -
List item
Orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid for: Treatment of systemic sclerosis
Date of first decision: 12/01/2017, Positive, Last updated: 11/08/2020 -
List item
Orphan designation: (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid- for: Treatment of cystic fibrosis
Date of first decision: 14/10/2016, Positive, Last updated: 11/08/2020 -
List item
Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid for: Treatment of dermatomyositis
Positive, Last updated: 29/07/2020 -
List item
Orphan designation: 4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1 (pridopidine hydrochloride) for: Treatment of Huntington’s disease
Date of first decision: 20/06/2005, Positive, Last updated: 28/07/2020