Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2014-04-29 | 3-bromopyruvate | PreScience Labs (USA - MD) | pancreatic cancer |
Granting of the orphan status in the US |
2017-12-11 | 3-pentylbenzenacetic acid sodium salt | ProMetic Life Sciences (Canada) |
|
Granting of a Fast Track status |
2017-03-23 | dinutuximab | United Therapeutics (USA - MD) | first-line therapy for pediatric patients with high-risk neuroblastoma | Withdrawal of a market application in the EU |
2015-03-19 | 5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3' | CTI Clinical Trial and Consulting Services Europe GmbH (Germany) | Alport syndrome | Granting of the orphan status in the EU |
2015-06-18 | 6-ethoxy-7-methoxy-2-(2-methylsulfanylphenyl)-3,1-benzoxazin-4-one | Sixera Pharma (Sweden) | Netherton syndrome |
Granting of the orphan status in the US |
2015-03-19 | human plasma-derived alpha-1 proteinase inhibitor | Richardson Associates Regulatory Affairs (UK) | graft-versus-host disease |
Granting of the orphan status in the EU |
2015-03-19 | recombinant human club cell 10 KDa protein | RLM Consulting (Belgium) | prevention of bronchopulmonary dysplasia | Granting of the orphan status in the EU |
2015-03-19 | tideglusib | QRC Consultants (UK) | fragile X syndrome |
Granting of the orphan status in the EU |
2015-11-02 | [5-(5-chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3- ylmethyl)-amine hydrochloride | Daiichi Sankyo (Japan) Plexxikon (USA - CA) | tenosynovial giant cell tumour, localised and diffuse type |
Granting of the Breakthrough Therapy status |
2015-03-19 | adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene | Gauchers Association (UK) | Gaucher disease |
Granting of the orphan status in the EU |
2015-12-29 | chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA | Isis Pharmaceuticals (USA - CA) now Ionis Pharmaceuticals (USA - CA) | Huntington’s disease |
Granting of the orphan status in the US |
2015-03-19 | enoxacin | Impasara (UK) | amyotrophic lateral sclerosis |
Granting of the orphan status in the EU |
2015-03-19 | ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a COL17A1-encoding retroviral vector | Chiesi Farmaceutici (Italy) | epidermolysis bullosa | Granting of the orphan status in the EU |
2015-07-01 | Gallium Ga 68-edotreotide | Advanced Accelerator Applications (France) | diagnosis of gastro-entero-pancreatic neuroendocrine tumours |
Granting of the orphan status in the US |
2015-03-19 | humanised anti-folate receptor 1 monoclonal antibody conjugated to maytansinoid DM4 | ImmunoGen Europe (UK) Immunogen (USA - MA) | ovarian cancer |
Granting of the orphan status in the EU |
2015-03-19 | lenvatinib | Eisai (Japan) | hepatocellular carcinoma |
Granting of the orphan status in the EU |
2015-03-19 | melphalan flufenamide | Oncopeptides (Sweden) | plasma cell myeloma/multiple myeloma |
Granting of the orphan status in the EU |
2016-08-25 | recombinant, fully-human, IgG4 immunoglobulin with antigen-binding specificity for human vascular adhesion protein-1 (VAP-1) | Biotie Therapies (Finland) | primary sclerosing cholangitis |
Granting of the orphan status in the US |
2017-09-05 | trientine tetrahydrochloride | GMP Orphan (France) Voisin Consulting (France) | Wilson disease | Granting of a Market Authorisation in the EU |
2015-03-19 | sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2] dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a]purin-9-one | Universitätsklinikum Tübingen (Germany) | retinitis pigmentosa |
Granting of the orphan status in the EU |
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