For help on how to get the results you want, see our search tips.
2361 results
Categories
Human Remove Human filter
-
List item
Orphan designation: 5-aminolevulinic acid for: Treatment of glioma
Date of first decision: 12/01/2017, Positive, Last updated: 08/12/2020 -
List item
Orphan designation: Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor for: Treatment of retinitis pigmentosa
Date of first decision: 24/01/2013, Positive, Last updated: 07/12/2020 -
List item
Orphan designation: L-cysteine, L-leucyl-L-alpha-glutamyl-L-alpha-glutamyl-L-lysyl-L-lysylglycyl-L-asparaginyl-L-tyrosyl-L-valyl-L-valyl-L-threonyl-L-alpha-aspartyl-L-histidyl-S-[1-[(4-carboxycyclohexyl)methyl]-2,5-dioxo-3-pyrrolidinyl]-complex with keyhole limpet haemocyanin for: Treatment of glioma
Date of first decision: 27/10/2011, Withdrawn, Last updated: 04/12/2020 -
List item
Orphan designation: Adeno-associated viral vector serotype Anc80 containing the truncated human ATP7B gene under the control of the human alpha-1 antitrypsin promoter for: Treatment of Wilson's disease
Date of first decision: 23/08/2017, Withdrawn, Last updated: 03/12/2020 -
List item
Orphan designation: Sodium (2R,3S,5R)-5-(4-amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5-(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetrahydrofuran-2-yl)methyl phosphate (guadecitabine) for: Treatment of acute myeloid leukaemia
Date of first decision: 14/12/2015, Withdrawn, Last updated: 01/12/2020 -
List item
Orphan designation: Recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene for: Treatment of Leber's congenital amaurosis
Date of first decision: 16/10/2017, Positive, Last updated: 30/11/2020 -
List item
Orphan designation: Recombinant human bone morphogenetic protein 4 for: Treatment of glioma
Date of first decision: 15/10/2014, Positive, Last updated: 30/11/2020 -
List item
Orphan designation: Obiltoxaximab for: Treatment of anthrax
Date of first decision: 24/08/2018, Positive, Last updated: 27/11/2020 -
List item
Orphan designation: Picropodophyllin for: Treatment of glioma
Date of first decision: 23/08/2017, Positive, Last updated: 27/11/2020 -
List item
Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran) for: Treatment of primary hyperoxaluria
Date of first decision: 21/03/2016, Positive, Last updated: 25/11/2020 -
List item
Orphan designation: Hydroxychloroquine for: Treatment of antiphospholipid syndrome
Date of first decision: 12/01/2017, Positive, Last updated: 24/11/2020 -
List item
Orphan designation: synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker (zilucoplan) for: Treatment of paroxysmal nocturnal haemoglobinuria
Date of first decision: 14/10/2016, Positive, Last updated: 24/11/2020 -
List item
Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease
Date of first decision: 09/08/2012, Positive, Last updated: 24/11/2020 -
List item
Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of first decision: 21/03/2012, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Acalabrutinib for: Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Date of first decision: 21/03/2016, Withdrawn, Last updated: 11/11/2020 -
List item
Orphan designation: Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B- for: Treatment of haemophilia B
Date of first decision: 14/12/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Triheptanoin for: Treatment of carnitine palmitoyltransferase II deficiency
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Triheptanoin for: Treatment of mitochondrial trifunctional protein deficiency
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Triheptanoin for: Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Triheptanoin for: Treatment of very long-chain acyl-CoA dehydrogenase deficiency
Date of first decision: 19/06/2015, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (pariglasgene brecaparvovec) for: treatment of glycogen storage disease type Ia
Date of first decision: 18/11/2016, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase (avalotcagene ontaparvovec) for: Treatment of ornithine transcarbamylase deficiency
Date of first decision: 21/03/2016, Positive, Last updated: 11/11/2020 -
List item
Orphan designation: Setmelanotide for: Treatment of Prader-Willi syndrome
Date of first decision: 27/06/2016, Positive, Last updated: 09/11/2020 -
List item
Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency
Date of first decision: 14/07/2016, Positive, Last updated: 09/11/2020 -
List item
Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency
Date of first decision: 19/11/2018, Positive, Last updated: 09/11/2020