Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2015-10-09 | 2-chloro-N6-(3-iodobenzyl) adenosine-5\'-N-methyluronamide | PBS Regulatory Consulting Group (USA - MA) | hepatocellular carcinoma |
Granting of the orphan status in the EU |
2015-09-03 | lebrikizumab | Genentech, a member of Roche Group (USA - CA - Switzerland) | idiopathic pulmonary fibrosis |
Granting of the orphan status in the US |
2015-10-09 | highly purified formulation of Staphylococcus aureus protein A | Coté Orphan Consulting UK (UK) | immune thrombocytopenia/immune thrombocytopenic purpura |
Granting of the orphan status in the EU |
2015-11-19 | genetic fusion of an analogue of Vasoactive Intestinal Peptide (VIP) with its ELP biopolymer | PhaseBio Pharmaceuticals (USA - PA) | cardiomyopathy associated with dystrophinopathies; Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy BMD), and X-linked dilated cardiomyopathy (XL-dCMP) |
Granting of the orphan status in the US |
2015-11-18 | 4-[(7-Methoxy-2,3-dihydro-1,4-benzothiazepin-4(5H)-yl)methyl]benzoic acid, hemifumarate | Armgo Pharma (USA - NY) | Duchenne muscular dystrophy (DMD) |
Granting of the orphan status in the US |
2017-04-18 | autologous T Cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | Novartis (Switzerland) | diffuse large B-cell lymphoma, relapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who have failed two or more prior therapies | Granting of the Breakthrough Therapy status |
2015-09-11 | daratumumab | Janssen Research & Development, a J&J company (USA - NJ) | diffuse large B-cell lymphoma |
Granting of the orphan status in the US |
2015-12-08 | non-replicating recombinant adeno-associated virus (AAV) vector of serotype 9 expressing human iduronate-2-sulfatase | RegenXBio (USA - MD) | mucopolysaccharidosis type II
|
Granting of a patent |
2015-05-13 | 2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol- 6-yl)acetamide | Flatley Discovery Lab (USA - MA) | cystic fibrosis |
Granting of the orphan status in the US |
2016-10-18 | (6aR,10aR)-3-(1,1-dimethylheptyl)-6a,7,10,10a-tetrahydro-1-hydroxy-6,6-dimethyl-6H-Dibenzo(b,d)pyran-9-carboxylic acid | Corbus Pharmaceuticals (USA - MA) | cystic fibrosis |
Granting of the orphan status in the EU |
2016-05-30 | sodium nitrite and EDTA | Arch Biopartners (Canada)/ Arch Bio Ireland | Pseudomonas aeruginosa pulmonary infections in patients with cystic fibrosis |
Granting of the orphan status in the EU |
2015-01-26 | tarextumab | OncoMed Pharmaceuticals (USA - CA) | pancreatic cancer |
Granting of the orphan status in the US |
2015-04-27 | propranolol and etodolac | Vicus Therapeutics (USA - NJ) | pancreatic cancer | Granting of the orphan status in the US |
2016-05-12 | (R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1Hbenzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea | Trio Medicines (UK) | gastro-entero-pancreatic neuroendocrine tumours |
Granting of the orphan status in the US |
2015-12-31 | adeno-associated viral vector expressing the human vascular endothelial growth factor gene | Integene International (USA - FL) | prevention of graft-versus-host disease (GVHD). |
Granting of the orphan status in the US |
2015-03-19 | 5,10,15,20-tetrakis(2,6-difluoro-3-N-methylsulfamoylphenyl)bacteriochlorin | Luzitin (Portugal) | biliary tract cancer |
Granting of the orphan status in the EU |
2015-12-16 | allogeneic mesenchymal stem cells product | Steminent Biotherapeutics (Taiwan - Canada) | polyglutamine spinocerebellar ataxia |
Granting of the orphan status in the US |
2015-12-09 | propagermanium and irbesartan | Dimerix Bioscience (Australia) | focal segmental glomerulosclerosis |
Granting of the orphan status in the US |
2015-12-14 | 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)methylphosphate | Otsuka Pharmaceutical (Japan) | acute myeloid leukaemia |
Granting of the orphan status in the EU |
2015-12-14 | variant of recombinant human fibroblast growth factor 19 | Diamond Pharma (UK) | primary sclerosing cholangitis |
Granting of the orphan status in the EU |
© 2024 Biopharmanalyses - Powered by Samacom+