For help on how to get the results you want, see our search tips.
2671 results
Medicine
Paediatric investigation plans Remove Paediatric investigation plans filter
Orphan designations Remove Orphan designations filter
Categories
Human Remove Human filter
-
List item
Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of designation: 18/12/2002, Positive, Last updated: 25/09/2023 -
List item
Orphan designation: 7-Beta-hydroxy cholesteryl-3-beta-oleate for: Treatment of glioma
Date of designation: 17/12/2010, Positive, Last updated: 22/09/2023 -
List item
Orphan designation: (S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrrol-2(1H)-yl)-3-hydroxy-2-phenylpropan-1-one for: Treatment of sickle cell disease
Date of designation: 19/10/2020, Positive, Last updated: 22/09/2023 -
List item
Orphan designation: Talquetamab for: Treatment of multiple myeloma
Date of designation: 20/08/2021, Positive, Last updated: 21/09/2023 -
List item
Orphan designation: Celecoxib, Ciprofloxacin for: Treatment of amyotrophic lateral sclerosis
Date of designation: 06/01/2021, Positive, Last updated: 19/09/2023 -
List item
Orphan designation: 3-{[2,3,5,6-tetrafluoro-3'-(trifluoromethoxy)biphenyl-4-yl]carbamoyl}thiophene-2-carboxylic acid for: Treatment of non-infectious uveitis
Date of designation: 19/06/2015, Positive, Last updated: 18/09/2023 -
List item
Orphan designation: N-(5-tert-Butylisoxazol-3-yl)-N'-{4-[7-(2-(morpholin-4-yl)ethoxy) imidazo[2,1-b][1,3]benzothiazol-2-yl]phenyl}urea di-hydrochloride salt for: Treatment of acute myeloid leukaemia
Date of designation: 23/03/2009, Positive, Last updated: 15/09/2023 -
List item
Orphan designation: Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-methoxy, ether with N-[[[2-[[6-[[1-[3-[[3-(2,3-dihydroxypropoxy)propyl]amino]-3-oxopropyl]-2,5-dioxo-3-pyrrolidinyl]thio]hexyl]amino]ethyl]amino]carbonyl]-2-methylalanyl-teriparatide (2:1) for: Treatment of hypoparathyroidism
Date of designation: 19/10/2020, Positive, Last updated: 15/09/2023 -
List item
Orphan designation: Dabrafenib mesylate for: Treatment of glioma
Date of designation: 09/12/2020, Positive, Last updated: 15/09/2023 -
List item
Orphan designation: zilucoplan for: Treatment of myasthenia gravis
Date of designation: 18/07/2022, Positive, Last updated: 15/09/2023 -
List item
Orphan designation: Asunercept for: Treatment of myelodysplastic syndromes
Date of designation: 23/08/2017, Positive, Last updated: 15/09/2023 -
List item
Orphan designation: Recombinant fusion protein consisting of the extracellular portion of CD95 fused to the Fc part of a human IgG1 molecule for: Treatment of glioma
Date of designation: 28/01/2010, Positive, Last updated: 15/09/2023 -
List item
Orphan designation: Recombinant fusion protein consisting of the extracellular portion of CD95 fused to the Fc part of a human IgG1 molecule for: Prevention of graft-versus-host disease
Date of designation: 31/10/2006, Positive, Last updated: 15/09/2023 -
List item
Orphan designation: 2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib) for: Treatment of medullary thyroid carcinoma
Date of designation: 19/02/2021, Positive, Last updated: 13/09/2023 -
List item
Orphan designation: Entinostat for: Treatment of Hodgkin's lymphoma
Date of designation: 10/06/2010, Positive, Last updated: 13/09/2023 -
List item
Orphan designation: Mixture of seven synthetic fragments consisting of p21 RAS peptides for: Treatment of pancreatic cancer
Date of designation: 05/08/2011, Positive, Last updated: 13/09/2023 -
List item
Orphan designation: Enzastaurin hydrochloride for: Treatment of diffuse large B-cell lymphoma
Date of designation: 20/03/2007, Withdrawn, Last updated: 12/09/2023 -
List item
Orphan designation: Risdiplam for: Treatment of spinal muscular atrophy
Date of designation: 26/02/2019, Withdrawn, Last updated: 12/09/2023 -
List item
Orphan designation: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of designation: 17/01/2018, Withdrawn, Last updated: 12/09/2023 -
List item
Orphan designation: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene (devafidugene civaparvovec) for: Treatment of mucopolysaccharidosis type I
Date of designation: 17/01/2018, Withdrawn, Last updated: 12/09/2023 -
List item
Orphan designation: Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for: Treatment of Fanconi anaemia type A
Date of designation: 17/12/2010, Positive, Last updated: 11/09/2023 -
List item
Orphan designation: 2',3',5'-Tri-O-acetyluridine for: Treatment of 5-fluorouracil overdose
Date of designation: 15/05/2009, Positive, Last updated: 11/09/2023 -
List item
Orphan designation: 4-Amino-(6R,S)-5,6,7,8-tetrahydro-L-biopterin dihydrochloride (ronopterin) for: Treatment of moderate and severe traumatic brain injury
Date of designation: 28/08/2006, Positive, Last updated: 11/08/2023 -
List item
Orphan designation: Adeno-associated virus serotype HSC 15 expressing human iduronate 2-sulfatase (Bevufenogene nofeparvovec) for: Treatment of mucopolysaccharidosis type II (Hunter Syndrome)
Date of designation: 16/03/2022, Withdrawn, Last updated: 08/08/2023 -
List item
Orphan designation: Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase for: Treatment of phenylalanine hydroxylase deficiency
Date of designation: 14/12/2018, Withdrawn, Last updated: 08/08/2023