Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2017-02-27 | soluble recombinant human fibroblast growth factor receptor 3 | Therachon (France) | achondroplasia | Granting of the orphan status in the EU |
2017-02-27 | tauroursodeoxycholic acid | Bruschettini (Italy) | amyotrophic lateral sclerosis | Granting of the orphan status in the EU |
2017-02-27 | thalidomide | PlumeStars (Italy) | hereditary haemorrhagic telangiectasia | Granting of the orphan status in the EU |
2017-02-27 | vemurafenib | Groupe d’étude des histiocytoses (France) | Erdheim-Chester disease | Granting of the orphan status in the EU |
2017-03-20 | acetylleucine | IntraBio (UK) | Niemann-Pick disease | Granting of the orphan status in the EU |
2017-03-20 | antisense oligonucleotide targeting the USH2A gene | ProQR Therapeutics (The Netherlands) | retinitis pigmentosa | Granting of the orphan status in the EU |
2017-03-20 | inebilizumab | AstraZeneca (UK) | neuromyelitis optica spectrum disorders | Granting of the orphan status in the EU |
2017-03-20 | ketoconazole | Grupo Español de Tumores Huérfanos e Infrecuentes (GETHI) (Spain) | granulosa cell tumours | Granting of the orphan status in the EU |
2017-03-20 | megestrol acetate | Grupo Español de Tumores Huérfanos e Infrecuentes (GETHI) (Spain) | granulosa cell tumours | Granting of the orphan status in the EU |
2017-03-20 | (3'R,4'S,5'R)-N-[(3R,6S)-6-carbamoyltetrahydro-2H-pyran-3-yl]-6''-chloro-4'-(2-chloro-3- fluoropyridin-4-yl)-4,4-dimethyl-2''-oxo-1'',2''-dihydrodispiro[cyclohexane-1,2'-pyrrolidine-3',3''- indole]-5'-carboxamide mono(4-methylbenzenesulfonate) monohydrate | Daiichi Sankyo (Japan) | soft tissue sarcoma | Granting of the orphan status in the EU |
2017-03-20 | adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene | Freeline Therapeutics (UK) | Fabry disease | Granting of the orphan status in the EU |
2017-03-20 | adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase | Dr Julien Baruteau (UK) | ornithine transcarbamylase deficiency | Granting of the orphan status in the EU |
2017-03-20 | adeno-associated viral vector serotype rh.10 expressing beta-galactosidase | Lysogene (France) | GM1 gangliosidosis | Granting of the orphan status in the EU |
2017-03-20 | allogeneic ex–vivo-expanded umbilical cord blood-derived haematopoietic CD34+ progenitor cells and allogeneic non-expanded umbilical cord blood-derived haematopoietic mature myeloid and lymphoid cells | Regulatory Resources Group (UK) | haematopoietic stem cell transplantation | Granting of the orphan status in the EU |
2017-03-20 | poly-cyclodextrin-bis-cysteine-PEG3400-camptothecin-conjugate | Viadoc Business Solutions (UK) | ovarian cancer | Granting of the orphan status in the EU |
2017-03-20 | phosphoinositide 3-kinase gamma peptide | Kither Biotech (Italy) | cystic fibrosis | Granting of the orphan status in the EU |
2017-03-20 | autologous adipose tissue-derived mesenchymal stem cells | SPC GmbH (Germany) | thromboangiitis obliterans (Buerger's disease) | Granting of the orphan status in the EU |
2017-04-20 | thymidine and deoxycytidine | Vall d'Hebron Institute of Research (Spain) | mitochondrial DNA depletion syndrome, myopathic form | Granting of the orphan status in the EU |
2017-04-20 | N-[(1R)-1-phenylethyl]-6-{1H-pyrazolo[3,4-d]pyrimidin-4-yl}quinazolin-2-amine | Sentinel Oncology (UK) | fragile X syndrome | Granting of the orphan status in the EU |
2017-04-20 | (S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8- diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester | Biological Consulting Europe (UK) | pulmonary arterial hypertension | Granting of the orphan status in the EU |
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