For help on how to get the results you want, see our search tips.
22 results
Categories
Human Remove Human filter
-
List item
Orphan designation: Allogeneic umbilical cord tissue-derived mesenchymal stromal cells ex vivo expanded for: Prevention of bronchopulmonary dysplasia
Date of designation: 21/08/2020, Positive, Last updated: 24/11/2020 -
List item
Orphan designation: Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Adeno-associated virus serotype 2/8 vector containing the human PDE6A gene for: Treatment of retinitis pigmentosa
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate for: Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Allogeneic hepatoblastoma cells encapsulated in alginate, ex vivo expanded for: Treatment of acute liver failure
Date of designation: 21/08/2020, Positive, Last updated: 24/06/2021 -
List item
Orphan designation: 2-(2-(18F)fluoropyridin-4-yl)-9H-pyrrolo[2,3-b:4,5-c']dipyridine for: Diagnosis of progressive supranuclear palsy
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Humanised IgG1 monoclonal antibody against human eotaxin-2 for: Treatment of primary sclerosing cholangitis
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: pentosan polysulfate sodium for: Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Date of designation: 21/08/2020, Positive, Last updated: 15/12/2022 -
List item
Orphan designation: Protein-based delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli for: Prevention of haemolytic uraemic syndrome
Date of designation: 21/08/2020, Positive, Last updated: 22/06/2022 -
List item
Orphan designation: Allogeneic T-cell precursors, mobilised peripheral blood-derived, ex vivo cultured for: Treatment in haematopoietic stem cell transplantation
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Dextran sulfate low molecular weight for: Treatment of amyotrophic lateral sclerosis
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate for: Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Copper histidinate for: Treatment of Menkes disease
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Adeno-associated viral vector serotype 3B encoding shortened human ATP7B for: Treatment of Wilson's disease
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: 6-((3S,4S)-4-methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one (Tovinontrine) for: Treatment of sickle cell disease
Date of designation: 21/08/2020, Withdrawn, Last updated: 08/11/2022 -
List item
Orphan designation: Trehalose for: Treatment of mucopolysaccharidosis type III (Sanfilippo syndrome)
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: 3-(((1S,2S,3R)-2,3-difluoro-1-hydroxy-7-(methylsulfonyl)-2,3-dihydro-1H-inden-4-yl)oxy)-5-fluorobenzonitrile for: Treatment of von Hippel-Lindau disease
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Venglustat for: Treatment of GM2 gangliosidosis
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta for: Treatment of Gaucher disease
Date of designation: 21/08/2020, Positive, Last updated: 18/05/2022 -
List item
Orphan designation: Autologous T cells transduced with lentiviral vector containing a tandem chimeric antigen receptor directed against CD20 and CD19 for: Treatment of diffuse large B-cell lymphoma
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Human frataxin fused to TAT cell-penetrating peptide for: Treatment of Friedreich’s ataxia
Date of designation: 21/08/2020, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Infigratinib for: Treatment of cholangiocarcinoma
Date of designation: 21/08/2020, Withdrawn, Last updated: 08/11/2022