Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-02-02 | osimertinib - N-(2-{2-dimethylaminoethyl-methylamino}-4-methoxy-5-{[4-(1-methylindol-3-yl)pyrimidin-2-yl]amino}phenyl)prop-2-enamide mesylate salt | AstraZeneca (UK) | epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer |
Granting of a Market Authorisation in the EU |
2015-01-26 | (S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1- f][1,2,4]triazine-5-carbonitrile | Almirall (Spain) | pemphigus vulgaris |
Granting of the orphan status in the US |
2014-10-15 | (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride | Khondrion (The Netherlands) | Leigh syndrome |
Granting of the orphan status in the EU |
2016-04-01 | mercaptamine hydrochloride or cysteamine hydrochloride | Lucane Pharma (France) | cystinosis |
Refusal of a Market Authorisation in the EU |
2014-10-15 | cultured allogeneic corneal limbal stem cells | Scottish National Blood Transfusion Service (UK) | limbal stem cell deficiency |
Granting of the orphan status in the EU |
2014-10-15 | immunoglobulin G1, anti-(human tumour-associated calcium signal transducer 2)(human-Mus musculus monoclonal hRS7 heavy chain), disulfide with human-Mus musculus monoclonal hRS7 k- chain, dimer, hexakis(thioether) with (4S)-4-[[[[4-[[(2S)-2-(4-aminobutyl)-2-[[2-[2-[[26-[4-[[[[4- [(3-mercapto-2,5-dioxo-1-pyrrolidinyl)methyl]cyclohexyl]carbonyl]amino]methyl]-1H-1,2,3-triazol- 1-yl]-3,6,9,12,15,18,21,24-octaoxahexacos-1-yl]amino]-2-oxoethoxy]acetyl]amino]-1- oxoethyl]amino]phenyl]methoxy]carbonyl]ox | Immunomedics (USA - NJ) | pancreatic cancer |
Granting of the orphan status in the EU |
2014-10-15 | osilodrostat | Novartis (Switzerland) | Cushing's syndrome |
Granting of the orphan status in the EU |
2014-10-15 | Oxalobacter formigenes strain HC-1 | OxThera (Sweden) | short bowel syndrome |
Granting of the orphan status in the EU |
2015-12-09 | (3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one | Centre National de la Recherche Scientifique (CNRS) (France) | fragile X syndrome |
Granting of the orphan status in the US |
2014-10-15 | acamprosate calcium | Real Regulatory Limited (Ireland) Confluence Pharmaceuticals (USA - IN) | fragile X syndrome |
Granting of the orphan status in the EU |
2014-10-15 | adeno-associated viral vector serotype 8 containing the human UGT1A1 gene | Genethon (France) | Crigler-Najjar syndrome |
Granting of the orphan status in the EU |
2014-10-15 | glucagon | S-Cubed (Denmark) | congenital hyperinsulinism |
Granting of the orphan status in the EU |
2014-10-15 | nitric oxide | PD Dr.med. Joachim Riethmüller | cystic fibrosis |
Granting of the orphan status in the EU |
2014-10-15 | pyridoxal 5\'-phosphate | Great Ormond Street Hospital Foundation Trust (UK) | pyridoxamine 5\'-phosphate oxidase deficiency |
Granting of the orphan status in the EU |
2014-10-15 | recombinant human bone morphogenetic protein 4 | Stemgen (Italy) | glioma |
Granting of the orphan status in the EU |
2014-10-15 | recombinant human insulin receptor monoclonal antibody-fused-alpha-L-iduronidase | Voisin Consulting (France) | mucopolysaccharidosis type I |
Granting of the orphan status in the EU |
2018-04-30 | burosumab -recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 | Ultragenyx Pharmaceutical (USA - CA) Kyowa Hakko Kirin (Japan) | X- linked hypophosphataemia (XLH) | Product launch |
2016-04-07 | enzalutamide | Astellas Pharma (Japan) Medivation (USA - CA) | patients with metastatic castration-resistant prostate cancer (CRPC) who have not received chemotherapy | Granting of a Market Authorisation in the EU |
2014-11-19 | (3S)-1-azabicyclo[2.2.2]oct-3-yl {2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate | Genzyme (USA - MA), a Sanofi company (France) | Gaucher disease |
Granting of the orphan status in the EU |
2014-09-16 | humanized monoclonal antibodies hu1B7 and hu11E6 | Synthetic Biologics (USA - MD) | Bordetella pertussis infection |
Granting of a patent |
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