Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2012-04-04 | kifunensine | Généthon (France) | gamma sarcoglycanopathy | Granting of the orphan status in the EU |
2011-09-27 | dinaciclib | Merck&Co (USA) | chronic lymphocytic leukemia |
Granting of the orphan status in the US |
2012-04-04 | 20-pentaerythritol poly (oxy-1,2-ethanediyl)-carboxymethyl-glycinate-7-ethyl-10-hydroxycamptothecine 10-[1,4\'-bipiperidine]-1\'-carboxylate | Nektar Therapeutics UK Ltd (UK) | ovarian cancer | |
2017-03-30 | ixazomib - MLN9708 - 2,2'-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid | Takeda Global Research and Development Centre (UK) Millennium Pharmaceuticals (USA - MA) | multiple myeloma | Granting of a Market Authorisation in Japan |
2012-04-04 | NH2-Cys-Ser-Ser-Val-Thr-Ala-Trp-Thr-Thr-Gly-Cys-Gly-CONH2 | Pharmaxon (France) | traumatic spinal cord injury | |
2015-03-26 | macitentan | Actelion (Switzerland) | pulmonary arterial hypertension (PAH) |
Granting of a Market Authorisation in Japan |
2012-04-04 | heterologous human adult liver-derived stem cells | Fresenius Medical Care (Germany) | ornithine transcarbamylase deficiency |
|
2012-04-04 | genetically modified Lactococcus lactis bacteria containing the human trefoil factor 1 gene | ActoGeniX (Belgium) | prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy. |
|
2012-04-04 | eflornithine | Cancer Prevention Pharma (UK) | neuroblastoma | |
2012-04-04 | sinapultide, dipalmitoylphosphatidylcholine palmitoyl-oleoyl phosphatidylglycerol, sodium salt and palmitic acid | Pharm Research Associates (UK) | cystic fibrosis | |
2012-04-04 | mifepristone | Voisin Consulting (France) | hypercortisolism (Cushing’s syndrome) of endogenous origin | |
2016-07-15 | lenalidomide | Celgene (USA - NJ) | mantle cell lymphoma |
Granting of a Market Authorisation in the EU |
2012-04-04 | human platelet antigen-1a immunoglobulin | Prophylix Pharma (Norway) | fetal and neonatal alloimmune thrombocytopenia due to human platelet antigen-1a incompatibility | |
2012-04-04 | GILT-tagged recombinant human acid alpha glucosidase | BioMarin Europe (UK) | glycogen storage disease type II (Pompe’s disease) |
Granting of the orphan status in the EU |
2012-04-04 | brivanib alaninate | BMS (USA) | hepatocellular carcinoma |
Granting of the orphan status in the EU |
2012-04-04 | adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase gene | Institut Pasteur (France) | mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) | |
2012-04-04 | 1-(4-{4-amino-7-[1-(2-hydroxyethyl)-1H- pyrazol-4-yl]thieno[3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea | Abbott Laboratories (USA) | acute myeloid leukaemia | |
2012-04-04 | recombinant human minibody against complement component C5 | Adienne (Italy) | primary membranoproliferative glomerulonephritis | |
2012-04-04 | 2-hydroxyoleic acid | Lipopharma Therapeutics (Spain) | glioma | |
2012-04-04 | nanoliposomal irinotecan | Merrimack Pharmaceuticals UK (UK) | pancreatic cancer |
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