For help on how to get the results you want, see our search tips.
250 results
Medicine
European public assessment reports (EPAR) Remove European public assessment reports (EPAR) filter
Direct healthcare professional communication Remove Direct healthcare professional communication filter
Summaries of opinion Remove Summaries of opinion filter
Orphan designations Remove Orphan designations filter
Medicine type
Exceptional circumstances Remove Exceptional circumstances filter
Orphan medicine Remove Orphan medicine filter
Orphan designation status
Positive Remove Positive filter
-
List item
Orphan designation: Sodium benzoate for: Treatment of hyperargininaemia
Date of designation: 14/07/2016, Positive, Last updated: 06/09/2016 -
List item
Orphan designation: Pegylated recombinant phenylalanine ammonia lyase (pegvaliase) for: Treatment of hyperphenylalaninaemia
Date of designation: 28/01/2010, Positive, Last updated: 29/05/2019 -
List item
Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of designation: 14/09/2007, Positive, Last updated: 26/03/2019 -
List item
Orphan designation: Sodium benzoate for: Treatment of lysinuric protein intolerance
Date of designation: 29/08/2016, Positive, Last updated: 03/10/2016 -
List item
Orphan designation: Gusperimus trihydrochloride for: Treatment of Wegener's granulomatosis
Date of designation: 29/03/2001, Positive, Last updated: 18/11/2011 -
List item
Orphan designation: Cysteamine hydrochloride for: Treatment of cystinosis
Date of designation: 15/10/2014, Positive, Last updated: 12/11/2014 -
List item
Orphan designation: Ethyl Eicosapentaenoate for: Treatment of Huntington’s disease
Date of designation: 29/12/2000, Positive, Last updated: 04/01/2006 -
List item
Orphan designation: idebenone for: Treatment of Friedreich's ataxia
Date of designation: 08/03/2004, Positive, Last updated: 11/04/2016 -
List item
Orphan designation: synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (Vutrisiran) for: Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis)
Date of designation: 25/05/2018, Positive, Last updated: 12/10/2022 -
List item
Orphan designation: Budesonide for: Treatment of eosinophilic oesophagitis
Date of designation: 05/08/2013, Positive, Last updated: 18/01/2018 -
List item
Orphan designation: NGR-human tumour necrosis factor for: Treatment of malignant mesothelioma
Date of designation: 03/06/2008, Positive, Last updated: 29/07/2008 -
List item
Orphan designation: mifepristone for: Treatment of hypercortisolism (Cushing's syndrome) of endogenous origin
Date of designation: 27/10/2011, Positive, Last updated: 18/03/2022 -
List item
Orphan designation: Anti epidermal growth factor receptor antibody h-R3 for: Treatment of glioma
Date of designation: 02/09/2004, Positive, Last updated: 12/10/2018 -
List item
Orphan designation: Levoglutamide for: Treatment of sickle cell disease
Date of designation: 04/07/2012, Positive, Last updated: 12/07/2019 -
List item
Orphan designation: sinapultide, Dipalmitoylphosphatidylcholine, palmitoyloleoyl phosphatidylglycerol, palmitic acid for: Respiratory distress syndrome in premature neonates of less than 32 weeks of gestational age
Date of designation: 29/07/2004, Positive, Last updated: 22/05/2019 -
List item
Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 16/01/2014, Positive, Last updated: 03/12/2021 -
List item
Orphan designation: Mexiletine hydrochloride for: Treatment of myotonic disorders
Date of designation: 19/11/2014, Positive, Last updated: 13/06/2018 -
List item
Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of designation: 12/03/2013, Positive, Last updated: 09/02/2016 -
List item
Orphan designation: Human heterologous liver cells for: Treatment of ornithine-transcarbamylase deficiency
Date of designation: 14/09/2007, Positive, Last updated: 16/08/2016 -
List item
Orphan designation: Rubitecan for: Treatment of pancreatic cancer
Date of designation: 10/06/2003, Positive, Last updated: 23/06/2003 -
List item
Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrom
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
List item
Orphan designation: (E)-(1S,4S,10S,21R)-7-[(Z)-ethylidene]-4,21-diisopropyl-2-oxa-12,13-dithia-5,8,20,23- tetraazabicyclo[8.7.6]tricos-16-ene-3,6,9,19,22-pentone for: Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)
Date of designation: 28/10/2005, Positive, Last updated: 12/10/2018 -
List item
Orphan designation: Pralatrexate for: Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)
Date of designation: 13/04/2007, Positive, Last updated: 07/04/2011 -
List item
Orphan designation: Sodium benzoate for: Treatment of citrullinaemia type 1
Date of designation: 14/07/2016, Positive, Last updated: 06/09/2016 -
List item
Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of designation: 23/04/2012, Positive, Last updated: 22/09/2020