For help on how to get the results you want, see our search tips.
117 results
Medicine type
Conditional approval Remove Conditional approval filter
Additional monitoring Remove Additional monitoring filter
Accelerated assessment Remove Accelerated assessment filter
Medicine
Orphan designations Remove Orphan designations filter
Paediatric investigation plans Remove Paediatric investigation plans filter
Orphan designation status
Positive Remove Positive filter
-
List item
Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for: Treatment of gastrointestinal stromal tumours
Date of first decision: 17/07/2017, Positive, Last updated: 22/05/2019 -
List item
Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of hyperargininaemia
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
List item
Orphan designation: Gallium (68Ga)-edotreotide for: Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Date of first decision: 19/03/2015, Positive, -
List item
Orphan designation: Recombinant human alpha-Mannosidase (velmanase alfa) for: Treatment of alpha-mannosidosis
Date of first decision: 26/01/2005, Positive, -
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: Gilteritinib for: Treatment of acute myeloid leukaemia
Date of first decision: 17/01/2018, Positive, Last updated: 08/11/2019 -
List item
Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of first decision: 24/07/2009, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of ornithine carbamoyltransferase deficiency
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
List item
Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
List item
Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of argininosuccinic aciduria
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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: Glyceryl tri-(4-phenylbutyrate) for: Treatment of citrullinaemia type 1
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
List item
Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline for: Treatment of tuberculosis
Date of first decision: 26/08/2005, Positive, Last updated: 01/04/2014 -
List item
Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of first decision: 26/03/2014, Positive, Last updated: 17/05/2019 -
List item
Orphan designation: Pegylated recombinant phenylalanine ammonia lyase (pegvaliase) for: Treatment of hyperphenylalaninaemia
Date of first decision: 28/01/2010, Positive, Last updated: 29/05/2019 -
List item
Orphan designation: Recombinant IgG degrading enzyme of Streptococcus pyogenes (imlifidase) for: Prevention of graft rejection following solid organ transplantation
Date of first decision: 12/01/2017, Positive, Last updated: 28/08/2020 -
List item
Orphan designation: Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel) for: Treatment of diffuse large B-cell lymphoma
Date of first decision: 14/10/2016, Positive, Last updated: 13/06/2018 -
List item
Orphan designation: [Gly2]-recombinant human glucagon-like peptide for: Treatment of short bowel syndrome
Date of first decision: 12/12/2001, Positive, Last updated: 15/08/2016 -
List item
Orphan designation: Adeno-associated viral vector expressing lipoprotein lipase for: Treatment of lipoprotein-lipase deficiency
Date of first decision: 09/03/2004, Positive, Last updated: 17/10/2013 -
List item
Orphan designation: budesonide for: Treatment of eosinophilic oesophagitis
Date of first decision: 05/08/2013, Positive, Last updated: 18/01/2018 -
List item
Orphan designation: Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel) for: Treatment of B-lymphoblastic leukaemia/lymphoma
Date of first decision: 29/04/2014, Positive, Last updated: 13/06/2018 -
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 primary mediastinal large B-cell lymphoma
Date of first decision: 09/10/2015, Positive, Last updated: 10/09/2018 -
List item
Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of first decision: 05/08/2013, Positive, Last updated: 20/11/2019 -
List item
Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of first decision: 30/11/2007, Positive, Last updated: 11/07/2014 -
List item
Orphan designation: idebenone for: Treatment of Leber's hereditary optic neuropathy
Date of first decision: 16/02/2007, Positive, Last updated: 11/04/2016