For help on how to get the results you want, see our search tips.
222 results
Categories
Human Remove Human filter
Medicine
Direct healthcare professional communication Remove Direct healthcare professional communication filter
Orphan designations Remove Orphan designations filter
Medicine type
Orphan medicine Remove Orphan medicine 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: Masitinib mesilate for: Treatment of pancreatic cancer
Date of first decision: 28/10/2009, Positive, -
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: 1, 1'-[1,4-Phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane for: Treatment to mobilize progenitor cells prior to stem cell transplantation
Date of first decision: 20/10/2004, Positive, Last updated: 18/08/2008 -
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: Decitabine for: Treatment of acute myeloid leukaemia
Date of first decision: 08/06/2006, Positive, Last updated: 19/09/2013 -
List item
Orphan designation: 1,3-Propanedisulfonic acid, disodium salt for: Treatment of systemic secondary amyloidosis
Date of first decision: 31/07/2001, 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: Carboxypeptidase G2 for: Adjunctive treatment in patients at risk of methotrexate toxicity
Date of first decision: 03/02/2003, Positive, Last updated: 02/04/2020 -
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: Caffeine citrate for: Prevention of bronchopulmonary dysplasia
Date of first decision: 11/04/2014, Positive, -
List item
Orphan designation: Mepolizumab for: Treatment of hypereosinophilic syndrome
Date of first decision: 29/07/2004, Withdrawn, Last updated: 23/10/2020 -
List item
Orphan designation: Treprostinil sodium for: Treatment of chronic thromboembolic pulmonary hypertension
Date of first decision: 08/02/2013, Positive, Last updated: 08/04/2020 -
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: Pirfenidone for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 16/11/2004, Positive, Last updated: 26/03/2015 -
List item
Orphan designation: Taliglucerase alfa for: Treatment of Gaucher disease
Date of first decision: 23/03/2010, Positive, Last updated: 10/03/2011 -
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 first decision: 19/11/2014, Positive, Last updated: 15/10/2020 -
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: Ivosidenib for: Treatment of acute myeloid leukaemia
Date of first decision: 12/12/2016, Positive, Last updated: 04/12/2019 -
List item
Orphan designation: Nanoliposomal irinotecan for: Treatment of pancreatic cancer
Date of first decision: 09/12/2011, Positive, Last updated: 17/12/2018 -
List item
Orphan designation: Adenovirus-mediated herpes-simplex-virus thymidine-kinase (HKSV-tk) gene for: Treatment of high-grade glioma with subsequent use of ganciclovir sodium
Date of first decision: 06/02/2002, Positive, Last updated: 15/08/2016 -
List item
Orphan designation: Isavuconazonium sulfate for: Treatment of invasive aspergillosis
Date of first decision: 04/07/2014, Positive, Last updated: 21/05/2019 -
List item
Orphan designation: Sirolimus for: Treatment of chronic non-infectious uveitis
Date of first decision: 30/08/2011, Positive, Last updated: 21/09/2011 -
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