For help on how to get the results you want, see our search tips.
1859 results
Medicine
Withdrawn applications Remove Withdrawn applications filter
Summaries of opinion Remove Summaries of opinion filter
Orphan designations Remove Orphan designations filter
Categories
Human Remove Human filter
Orphan designation status
Positive Remove Positive filter
-
List item
Orphan designation: recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein, Eladocagene exuparvovec for: Treatment of aromatic L-amino acid decarboxylase deficiency (updated)
Date of designation: 18/11/2016, Positive, Last updated: 10/08/2022 -
List item
Orphan designation: 2-Chloro-N6-(3-iodobenzyl)adenosine-5'-N-methyluronamide (namodenoson) for: Treatment of hepatocellular carcinoma (updated)
Date of designation: 09/10/2015, Positive, Last updated: 10/08/2022 -
List item
Orphan designation: Adenovirus associated viral vector serotype 4 containing the human RPE65 gene for: Treatment of retinitis pigmentosa (updated)
Date of designation: 14/11/2007, Positive, Last updated: 09/08/2022 -
List item
Orphan designation: Adenovirus associated viral vector serotype 4 containing the human RPE65 gene for: Treatment of Leber's congenital amaurosis (updated)
Date of designation: 22/10/2007, Positive, Last updated: 09/08/2022 -
List item
Orphan designation: adenovirus associated viral vector serotype 5 containing the human pde6β gene for: Treatment of retinitis pigmentosa (updated)
Date of designation: 19/06/2013, Positive, Last updated: 09/08/2022 -
List item
Orphan designation: Adeno-associated viral vector serotype 5 containing the human RLBP1 gene for: Treatment of retinitis pigmentosa (updated)
Date of designation: 14/10/2016, Positive, Last updated: 09/08/2022 -
List item
Orphan designation: Mazindol for: Treatment of narcolepsy (updated)
Date of designation: 09/10/2015, Positive, Last updated: 03/08/2022 -
List item
Orphan designation: Bilayer engineered collagen hydrogel-based skin graft composed of autologous keratinocytes and fibroblasts for: Treatment of partial deep dermal and full-thickness burns (updated)
Date of designation: 14/12/2015, Positive, Last updated: 03/08/2022 -
List item
Orphan designation: Pegylated recombinant arginine deiminase (pegargiminase) for: Treatment of malignant mesothelioma (updated)
Date of designation: 15/01/2015, Positive, Last updated: 02/08/2022 -
List item
Orphan designation: (S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate for: Treatment of carcinoid syndrome (updated)
Date of designation: 30/05/2016, Positive, Last updated: 28/07/2022 -
List item
Orphan designation: Recombinant human acid sphingomyelinase (also known as olipudase alfa) for: Treatment of Niemann-Pick disease (updated)
Date of designation: 24/06/2022, Positive, Last updated: 27/07/2022 -
List item
Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) for: Treatment of spinal muscular atrophy (updated)
Date of designation: 19/06/2015, Positive, Last updated: 25/07/2022 -
List item
Orphan designation: Sirolimus for: Treatment of tuberous sclerosis (updated)
Date of designation: 20/06/2017, Positive, Last updated: 19/07/2022 -
List item
Orphan designation: budesonide for: Treatment of primary IgA nephropathy (updated)
Date of designation: 18/11/2016, Positive, Last updated: 18/07/2022 -
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: 14/07/2022 -
List item
Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene for: Treatment of Wiskott-Aldrich syndrome
Date of designation: 06/06/2012, Positive, Last updated: 14/07/2022 -
List item
Orphan designation: autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the alpha-L-iduronidase cDNA for: Treatment of mucopolysaccharidosis type I
Date of designation: 26/10/2018, Positive, Last updated: 14/07/2022 -
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
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)
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
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
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
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
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
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
Date of designation: 12/10/2017, Positive, Last updated: 14/07/2022