Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-03-29 | 3-(5-Fluoro-benzofuran-3-yl)-4-(5-methyl-5H-[1,3]dioxolo[4,5-f]indol-7-yl)-pyrrole-2,5-dione | Actuate Therapeutics (USA - TX) | glioblastoma |
Granting of the orphan status in the US |
2016-02-11 | 6-{4-[l-(propan-2-yl)piperidin-4-yl]-1,4-diazepan-1-yl}-N-(pyridin-4-yl)pyridine-2-carboxamide | Upsher-Smith Laboratories (USA) | glioblastoma multiforme (GBM) |
Granting of the orphan status in the US |
2016-06-23 | 7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one*2HCl | Oncoceutics (USA - PA) | glioblastoma |
Granting of the orphan status in the US |
2016-05-18 | combination of disulfiram and copper gluconate | Cantex Pharmaceuticals (USA - FL) | glioblastoma multiforme |
Granting of the orphan status in the US |
2016-05-04 | human recombinant bone morphogenetic protein 4 | HyperStem Therapeutics (USA - CA) | glioblastoma multiforme |
Granting of the orphan status in the US |
2016-06-02 | methimazole and liothyronine sodium | Musli Thyropeutics (Israel) | glioblastoma multiforme |
Granting of the orphan status in the US |
2016-05-19 | ropidoxuridine (5-iodo-2-pyrimidinone-2'-deoxyribose) | EMEK (USA) | radiosensitizer to be used during radiation treatment of glioblastoma multiforme |
Granting of the orphan status in the US |
2017-09-19 | telotristat etiprate - (S)-ethyl2-amino-3-(4-(2-amino-6-((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate | Ipsen (France) Lexicon Pharmaceuticals (USA - TX) |
|
Granting of a Market Authorisation in the EU |
2016-07-20 | Zika Virus Real-time RT-PCR assay | Viracor-IBT Laboratories (USA - MO), a wholly-owned subsidiary of Eurofins Scientific (Luxembourg) | Zika virus infection |
Granting of a Market Authorisation in the US |
2017-07-20 | brodalumab | AstraZeneca (UK) Valeant Pharmaceuticals (Canada) Leo Pharma (Denmark) | moderate-to-severe plaque psoriasis | Granting of a Market Authorisation in the EU |
2016-08-29 | autologous mesenchymal stromal cells on a decellularised tracheal scaffold from a cadaveric donor | Videregen (UK) | tracheal stenosis |
Granting of the orphan status in the EU |
2017-09-14 | masitinib | AB Science (France) | severe systemic mastocytosis unresponsive to optimal symptomatic treatment | Negative opinion for the granting of a Market Authorisation in the EU |
2016-08-29 | recombinant protein derived from the saliva of the Ornithodoros moubata tick | Akari Therapeutics (USA - NY - UK) | paroxysmal nocturnal haemoglobinuria |
Granting of the orphan status in the EU |
2016-08-29 | sodium benzoate | Lucane Pharma (France) | ornithine translocase deficiency |
Granting of the orphan status in the EU |
2016-08-29 | sodium benzoate | Lucane Pharma (France) | lysinuric protein intolerance |
Granting of the orphan status in the EU |
2016-06-14 | triheptanoin | Vall d'Hebron Institute of Research (Spain) | McArdle’s disease (glycogen storage disease due to muscle glycogen phosphorylase deficiency) |
Positive opinion for the granting of the orphan status in the EU |
2016-08-29 | valproic acid | Vall d'Hebron Institute of Research (Spain) | McArdle’s disease (glycogen storage disease due to muscle glycogen phosphorylase deficiency) |
Granting of the orphan status in the EU |
2016-08-29 | zoledronic acid | Laboratorio Italiano Biochimico Farmaceutico Lisapharma (Italy) | glioma |
Granting of the orphan status in the EU |
2016-08-29 | 2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2- alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile | Galapagos (Belgium) | idiopathic pulmonary fibrosis |
Granting of the orphan status in the EU |
2016-08-29 | 2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]- phenyl}-2-oxo-acetamide | F2G (UK) | scedosporiosis |
Granting of the orphan status in the EU |
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