Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2014-01-15 | (6aS)-1,10-dimethoxy-6-methyl-5,6,6a,7-tetrahydro-4H-dibenzo[de,g]quinoline-2,9-diol | Valentia BioPharma (Spain) | dystrophic myotonia |
Granting of the orphan status in the EU |
2014-01-15 | amatuximab | Eisai Europe (UK) | malignant mesothelioma | |
2014-01-15 | N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino)phenyl)acrylamide benzenesulfonic acid salt | Celgene (USA - NJ) | chronic lymphocytic leukaemia / small lymphocytic lymphoma |
Granting of the orphan status in the EU |
2018-10-18 | pembrolizumab (MK-3475) | Merck&Co (USA - NJ) |
|
Positive opinion for the granting of a Market Authorisation in the EU |
2016-11-18 | budesonide | Pharmalink (Sweden) | primary IgA nephropathy | Granting of the orphan status in the EU |
2014-01-16 | idelalisib | Gilead Sciences (USA - CA) | refractory indolent non-Hodgkin’s lymphoma |
Acceptation for review of a NDA |
2015-06-25 | human heterologous liver cells | Cytonet (Germany - USA) | Urea Cycle Disorders (UCD) in children (carbamoylphosphate synthetase 1 deficiency, ornithine transcarbamylase deficiency, argininosuccinate synthetase deficiency (citrullinaemia type 1), argininosuccinate lyase deficiency (argininosuccinic aciduria) and arginase deficiency (hyperargininaemia) |
Negative opinion for the granting of a Market Authorisation in the EU |
2014-01-22 | 11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione | Synovo (Germany) | cystic fibrosis | |
2014-01-22 | cysteamine | Istituto Europeo per la Ricerca sulla Fibrosi Cistica – ONLUS (Italy) | cystic fibrosis |
Granting of the orphan status in the EU |
2014-01-22 | diacerein | Prof. Johann W. Bauer | epidermolysis bullosa |
Granting of the orphan status in the EU |
2014-03-26 | eculizumab | Alexion Europe (France - EMEA service center of Alexion Pharmaceuticals (USA - CT) | prevention of delayed graft function after solid organ transplantation |
Granting of the orphan status in the EU |
2014-02-19 | recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins | Roche (Switzerland) | prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection |
Granting of the orphan status in the US |
2014-01-22 | 3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl) amino]methyl}piperidin-1-yl]methanone | Neurolixis UK | Rett syndrome |
Granting of the orphan status in the EU |
2014-09-24 | gallium (68Ga) - pasireotide tetraxetan | OctreoPharm Sciences (Germany) | diagnosis of gastro-entero-pancreatic neuroendocrine tumours |
Granting of the orphan status in the US |
2014-01-22 | Asp-Arg-Val-Tyr-Ile-His-Pro | Gregory Fryer Associates Ltd (UK) | Duchenne muscular dystrophy (DMD) |
Granting of the orphan status in the EU |
2014-01-22 | autologous dendritic cells pulsed with tumour antigen-derived synthetic peptides (MAGE-1, HER-2, AIM-2, TRP-2, gp-100, and interleukin-13 receptor alpha) | Diamond BioPharm (UK) | glioma |
Granting of the orphan status in the EU |
2014-01-22 | N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2-fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide | Bionure Farma (Spain) | optic neuritis |
Granting of the orphan status in the EU |
2018-08-27 | volanesorsen - phosphorothioate oligonucleotide targeted to apolipoprotein C-III | Isis USA Ltd (UK) Isis Pharmaceuticals (USA - CA) Akcea Therapeutics (USA - MA) | familial chylomicronaemia syndrome (FCS) | Refusal of a Market Autorisation in the US |
2014-01-22 | pioglitazone | Minoryx Therapeutics (Spain) | adrenoleukodystrophy |
Granting of the orphan status in the EU |
2014-02-19 | recombinant human acid ceramidase | QOL Therapeutics EU Ltd (UK) | Farber disease |
Granting of the orphan status in the EU |
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