For help on how to get the results you want, see our search tips.
669 results
Medicine
Orphan designations Remove Orphan designations filter
Direct healthcare professional communication Remove Direct healthcare professional communication filter
Periodic safety update report single assessments Remove Periodic safety update report single assessments filter
Orphan designation status
Withdrawn Remove Withdrawn filter
-
List item
Orphan designation: Human autologous mesenchymal adult stem cells extracted from adipose tissue for: Treatment of anal fistula (updated)
Date of designation: 26/08/2005, Withdrawn, Last updated: 01/07/2022 -
List item
Orphan designation: Chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p for: Treatment of cutaneous T-cell lymphoma (updated)
Date of designation: 22/05/2017, Withdrawn, Last updated: 22/06/2022 -
List item
Orphan designation: Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat) for: Treatment of pulmonary arterial hypertension (updated)
Date of designation: 20/12/2007, Withdrawn, Last updated: 14/06/2022 -
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 primary sclerosing cholangitis (updated)
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
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 primary biliary cirrhosis (updated)
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
List item
Orphan designation: Trifarotene for: Treatment of autosomal recessive congenital ichthyosis (updated)
Date of designation: 24/03/2020, Withdrawn, Last updated: 07/06/2022 -
List item
Orphan designation: Inebilizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 20/03/2017, Withdrawn, Last updated: 19/05/2022 -
List item
Orphan designation: synthetic human hepcidin for: Treatment of sickle cell disease
Date of designation: 18/11/2016, Withdrawn, Last updated: 18/05/2022 -
List item
Orphan designation: Synthetic hepcidin for: Treatment of beta thalassaemia intermedia and major
Date of designation: 09/10/2015, Withdrawn, Last updated: 18/05/2022 -
List item
Orphan designation: Obeticholic acid for: Treatment of primary sclerosing cholangitis
Date of designation: 16/03/2014, Withdrawn, Last updated: 10/05/2022 -
List item
Orphan designation: N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) for: Treatment of Hodgkin's lymphoma
Date of designation: 14/09/2007, Withdrawn, Last updated: 10/05/2022 -
List item
Orphan designation: N- (2-Amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) for: Treatment of acute myeloid leukaemia
Date of designation: 31/01/2008, Withdrawn, Last updated: 04/05/2022 -
List item
Orphan designation: Lisocabtagene maraleucel for: Treatment of primary mediastinal large B-cell lymphoma
Date of designation: 19/11/2018, Withdrawn, Last updated: 04/05/2022 -
List item
Orphan designation: Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) for: Treatment of follicular lymphoma
Date of designation: 25/05/2018, Withdrawn, Last updated: 04/05/2022 -
List item
Orphan designation: Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 17/07/2017, Withdrawn, Last updated: 04/05/2022 -
List item
Orphan designation: Allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19 for: Treatment in haematopoietic stem cell transplantation
Date of designation: 27/06/2016, Withdrawn, Last updated: 07/04/2022 -
List item
Orphan designation: Rimiducid for: Treatment of graft-versus-host disease
Date of designation: 30/05/2016, Withdrawn, Last updated: 07/04/2022 -
List item
Orphan designation: Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol for: Treatment of cystic fibrosis
Date of designation: 30/08/2011, Withdrawn, Last updated: 06/04/2022 -
List item
Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel) for: Treatment of adrenoleukodystrophy
Date of designation: 06/06/2012, Withdrawn, Last updated: 04/04/2022 -
List item
Orphan designation: Adeno-associated virus serotype 9 expressing the cDNA for human MECP2 for: Treatment of Rett syndrome
Date of designation: 20/05/2021, Withdrawn, Last updated: 30/03/2022 -
List item
Orphan designation: Veledimex for: Treatment of glioma
Date of designation: 21/08/2019, Withdrawn, Last updated: 21/03/2022 -
List item
Orphan designation: Adenoviral vector serotype 5 encoding the human interleukin-12 p70 transgene under the control of activator ligand veledimex for: Treatment of glioma
Date of designation: 21/08/2019, Withdrawn, Last updated: 21/03/2022 -
List item
Orphan designation: Ponatinib hydrochloride for: Treatment of gastrointestinal stromal tumours
Date of designation: 15/01/2015, Withdrawn, Last updated: 18/03/2022 -
List item
Orphan designation: 3-pentylbenzeneacetic acid sodium salt for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 09/10/2015, Withdrawn, Last updated: 28/02/2022 -
List item
Orphan designation: 3-pentylbenzeneacetic acid sodium salt for: Treatment of Alström syndrome
Date of designation: 12/01/2017, Withdrawn, Last updated: 28/02/2022