For help on how to get the results you want, see our search tips.
4869 results
-
List item
Orphan designation: Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene for: Treatment of beta thalassaemia intermedia and major (updated)
Date of designation: 30/05/2016, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene for: Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome) (updated)
Date of designation: 10/06/2014, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: Autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene for: Treatment of X-linked chronic granulomatous disease (updated)
Date of designation: 09/02/2012, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy (updated)
Date of designation: 13/04/2007, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene for: Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency (updated)
Date of designation: 26/08/2005, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: Replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme for: Treatment of autosomal recessive congenital ichthyosis (updated)
Date of designation: 13/11/2019, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: genetically modified replication-incompetent herpes simplex virus-1 expressing collagen VII for: Treatment of epidermolysis bullosa (updated)
Date of designation: 16/04/2018, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: antisense oligonucleotide targeting exon 73 in the COL7A1 gene for: Treatment of epidermolysis bullosa (updated)
Date of designation: 12/10/2017, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: Darinaparsin for: Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated) (updated)
Date of designation: 15/04/2011, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of follicular lymphoma (updated)
Date of designation: 11/11/2015, Positive, Last updated: 12/07/2022 -
List item
Orphan designation: Melphalan flufenamide for: Treatment of plasma cell myeloma
Date of designation: 19/03/2015, Withdrawn, Last updated: 08/07/2022 -
List item
Orphan designation: adeno-associated virus serotype 9 vector containing human n-acetylgalactosamine-6-sulfate sulfatase gene for: Treatment of mucopolysaccharidosis type IVA (Morquio A Syndrome)
Date of designation: 09/01/2020, Positive, Last updated: 08/07/2022 -
List item
Orphan designation: Adeno-associated viral vector serotype 9 containing the human sulfamidase gene for: Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)
Date of designation: 21/06/2011, Positive, Last updated: 08/07/2022 -
List item
Orphan designation: Recombinant human monoclonal antibody binding to vascular adhesion protein-1 for: Treatment of primary sclerosing cholangitis
Date of designation: 19/03/2015, Positive, Last updated: 08/07/2022 -
List item
Orphan designation: Ilixadencel for: Treatment of gastrointestinal stromal tumours
Date of designation: 26/03/2021, Positive, Last updated: 08/07/2022 -
List item
Orphan designation: N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2-fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide for: Treatment of optic neuritis
Date of designation: 31/03/2014, Positive, Last updated: 05/07/2022 -
List item
Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide for: Treatment of neurofibromatosis type 2
Date of designation: 26/04/2012, Positive, Last updated: 05/07/2022 -
List item
Orphan designation: dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract) for: Treatment of epidermolysis bullosa
Date of designation: 23/02/2011, Positive, Last updated: 04/07/2022 -
List item
Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide for: Treatment of microscopic polyangiitis (avacopan)
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022 -
List item
Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide (avacopan) for: Treatment of granulomatosis with polyangiitis
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022 -
List item
Orphan designation: Human autologous mesenchymal adult stem cells extracted from adipose tissue for: Treatment of anal fistula
Date of designation: 26/08/2005, Withdrawn, Last updated: 01/07/2022 -
List item
Orphan designation: Setmelanotide for: Treatment of Alström syndrome
Date of designation: 09/01/2020, Positive, Last updated: 01/07/2022 -
List item
Orphan designation: Setmelanotide for: Treatment of Bardet-Biedl syndrome
Date of designation: 21/08/2019, Positive, Last updated: 01/07/2022 -
List item
Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency
Date of designation: 19/11/2018, Positive, Last updated: 01/07/2022 -
List item
Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency
Date of designation: 14/07/2016, Positive, Last updated: 01/07/2022