For help on how to get the results you want, see our search tips.
2782 results
-
List item
Orphan designation: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease
Date of designation: 24/04/2019, Withdrawn, Last updated: 13/04/2023 -
List item
Orphan designation: tripotassium citrate monohydrate, Potassium hydrogen carbonate for: Treatment of cystinuria
Date of designation: 09/01/2020, Positive, Last updated: 13/04/2023 -
List item
Orphan designation: arimoclomol citrate for: Treatment of Niemann-Pick's disease, type C
Date of designation: 19/11/2014, Positive, Last updated: 11/04/2023 -
List item
Orphan designation: (R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea (ceclazepide) for: Treatment of gastro-entero-pancreatic neuroendocrine tumours
Date of designation: 14/12/2015, Withdrawn, Last updated: 04/04/2023 -
List item
Orphan designation: Streptococcus pyogenes, group A, type 3, strain Su, inactivated for: Treatment of lymphatic malformations
Date of designation: 16/05/2022, Positive, Last updated: 04/04/2023 -
List item
Orphan designation: Tobramycin for: Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Date of designation: 17/03/2003, Positive, Last updated: 03/04/2023 -
List item
Orphan designation: Synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid (tofersen) for: Treatment of amyotrophic lateral sclerosis
Date of designation: 29/08/2016, Positive, Last updated: 03/04/2023 -
List item
Orphan designation: Humanised anti-alpha ν beta 6 monoclonal antibody for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 29/07/2014, Positive, Last updated: 03/04/2023 -
List item
Orphan designation: 2-(3-(4-(1H-Indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide-methane sulfonic acid salt for: Treatment of graft-versus-host disease
Date of designation: 17/10/2019, Positive, Last updated: 03/04/2023 -
List item
Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene for: Treatment of adenosine-deaminase-deficient severe combined immunodeficiency
Date of designation: 07/06/2013, Positive, Last updated: 03/04/2023 -
List item
Orphan designation: Iodine (131I) iobenguane for: Treatment of neuroblastoma
Date of designation: 31/01/2008, Positive, Last updated: 29/03/2023 -
List item
Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 16/12/2014, Positive, Last updated: 27/03/2023 -
List item
Orphan designation: Recombinant adeno-associated viral vector containing the human RPGR gene for: Treatment of retinitis pigmentosa caused by mutations in the RPGR gene
Date of designation: 30/05/2016, Positive, Last updated: 27/03/2023 -
List item
Orphan designation: (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidine-3-yl)chroman-2-carboxamide hydrochloride for: Treatment of maternally-inherited diabetes and deafness
Date of designation: 28/06/2019, Positive, Last updated: 27/03/2023 -
List item
Orphan designation: (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride for: Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes
Date of designation: 10/08/2015, Positive, Last updated: 27/03/2023 -
List item
Orphan designation: (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride for: Treatment of Leigh syndrome
Date of designation: 15/10/2014, Positive, Last updated: 27/03/2023 -
List item
Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel) for: Treatment of Wiskott-Aldrich syndrome
Date of designation: 06/06/2012, Positive, Last updated: 27/03/2023 -
List item
Orphan designation: Adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase for: Treatment of ornithine transcarbamylase deficiency
Date of designation: 20/03/2017, Positive, Last updated: 27/03/2023 -
List item
Orphan designation: Soluble recombinant human fibroblast growth factor receptor 3 for: Treatment of achondroplasia
Date of designation: 27/02/2017, Withdrawn, Last updated: 24/03/2023 -
List item
Orphan designation: miglustat for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 11/01/2019, Withdrawn, Last updated: 24/03/2023 -
List item
Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin gene for: Treatment of beta thalassaemia intermedia and major
Date of designation: 29/04/2009, Positive, Last updated: 24/03/2023 -
List item
Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of beta thalassaemia intermedia and major
Date of designation: 29/07/2014, Positive, Last updated: 21/03/2023 -
List item
Orphan designation: Autologous T-cells transduced with a lentiviral vector encoding a chimeric antigen receptor against CD7 for: Treatment of acute lymphoblastic leukaemia
Date of designation: 09/12/2022, Positive, Last updated: 14/03/2023 -
List item
Orphan designation: Panobinostat for: Treatment of multiple myeloma
Date of designation: 08/11/2012, Positive, Last updated: 13/03/2023 -
List item
Orphan designation: Lentiviral vector containing the human MYO7A gene for: Treatment of retinitis pigmentosa in Usher syndrome 1B
Date of designation: 23/03/2010, Positive, Last updated: 13/03/2023