For help on how to get the results you want, see our search tips.
2846 results
-
List item
Orphan designation: 68Ga-DOTA-pABzA-DIG-dPhe-Gln-Trp-Ala-Val-Gly-His-NHCH[(CH2-CH(CH3)2]2 for: Diagnosis of gastrointestinal stromal tumours
Date of designation: 12/12/2016, Positive, -
List item
Orphan designation: N'-(5-chloro-2-hydroxy-3-methylbenzylidene)-2,4-dihydroxybenzhydrazide for: Treatment of partial deep dermal and full-thickness burns
Date of designation: 22/09/2008, Positive, Last updated: 06/04/2011 -
List item
Orphan designation: Veliparib for: Treatment of ovarian cancer
Date of designation: 17/12/2010, Positive, Last updated: 29/05/2018 -
List item
Orphan designation: Cladribine for: Treatment of indolent non-Hodgkin's lymphoma
Date of designation: 18/09/2001, Expired, Last updated: 18/06/2014 -
List item
Orphan designation: (Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysyccinate for: Treatment of malignant gastrointestinal stromal tumours
Date of designation: 10/03/2005, Withdrawn, Last updated: 15/10/2009 -
List item
Orphan designation: Volasertib for: Treatment of acute myeloid leukaemia
Date of designation: 26/03/2014, Positive, -
List item
Orphan designation: 5,6,7,8-Tetrahydrobiopterin for: Treatment of hyperphenylalaninaemia
Date of designation: 02/10/2003, Positive, Last updated: 23/02/2007 -
List item
Orphan designation: Tralokinumab for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 08/11/2012, Withdrawn, -
List item
Orphan designation: Caffeine citrate for: Prevention of bronchopulmonary dysplasia
Date of designation: 11/04/2014, Positive, -
List item
Orphan designation: Volanesorsen sodium for: Treatment of familial partial lipodystrophy
Date of designation: 14/07/2016, Positive, Last updated: 23/05/2019 -
List item
Orphan designation: Recombinant human serum amyloid P for: Prevention of scarring post glaucoma filtration surgery
Date of designation: 09/10/2009, Withdrawn, -
List item
Orphan designation: Methotrexate for: Treatment of alkaptonuria
Date of designation: 29/08/2016, Positive, -
List item
Orphan designation: Vatreptacog alfa (activated) for: Treatment of haemophilia B
Date of designation: 09/08/2012, Withdrawn, -
List item
Orphan designation: Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys for: Treatment of high-altitude pulmonary oedema
Date of designation: 08/02/2013, Positive, -
List item
Orphan designation: 1,3-Propanedisulfonic acid, disodium salt for: Treatment of systemic secondary amyloidosis
Date of designation: 31/07/2001, Positive, -
List item
Orphan designation: milciclib maleate for: Treatment of malignant thymoma
Date of designation: 08/11/2012, Withdrawn, Last updated: 22/03/2021 -
List item
Orphan designation: Norursodeoxycholic acid for: Treatment of primary sclerosing cholangitis
Date of designation: 04/07/2014, Positive, -
List item
Orphan designation: 5-aminolevulinic acid for: Treatment of glioma
Date of designation: 12/01/2017, Positive, Last updated: 08/12/2020 -
List item
Orphan designation: Gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(2-7)disulfide for: Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Date of designation: 19/02/2014, Positive, -
List item
Orphan designation: Ethanol for: Treatment of congenital venous malformations
Date of designation: 09/03/2004, Withdrawn, Last updated: 18/01/2016 -
List item
Orphan designation: Diphenylcyclopropenone for: Treatment of alopecia totalis
Date of designation: 29/06/2006, Withdrawn, Last updated: 19/09/2013 -
List item
Orphan designation: 2-(2-Methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide for: Treatment of small cell lung cancer
Date of designation: 22/08/2014, Positive, -
List item
Orphan designation: Trebananib for: Treatment of ovarian cancer
Date of designation: 13/11/2013, Positive, -
List item
Orphan designation: Recombinant human C1-inhibitor for: Treatment of angioedema caused by C1 inhibitor deficiency
Date of designation: 11/05/2001, Withdrawn, Last updated: 18/11/2010 -
List item
Orphan designation: hydrocortisone for: Treatment of adrenal insufficiency
Date of designation: 20/03/2007, Positive, Last updated: 20/10/2020