Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2018-01-19 | adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and the human iduronate 2-sulfatase (hIDS) gene | Sangamo Therapeutics (USA - CA) previously known as Sangamo Biosciences | mucopolysaccharidosis type II (MPS II) | Granting of the orphan status in the EU |
2017-01-23 | metreleptin (analog of the human hormone leptin) | Novelion Therapeutics (Canada) |
|
Acceptation for review of a NDA |
2017-05-05 | edaravone | Mitsubishi Tanabe Pharma (Japan) | amyotrophic lateral sclerosis | Granting of a Market Authorisation in the US |
2017-06-27 | scAAV9.MeCP2.hCLN3, a self-complementary AAV serotype 9 expressing human CLN3 (hCLN3) | Abeona Therapeutics (USA - NY) | juvenile Batten disease | Granting of the orphan status in the US |
2017-01-12 | recombinant adeno-associated viral vector serotype 9 containing the human N-?- acetylglucosaminidase gene | Abeona Therapeutics (USA - NY) Sermes Planificacion (Spain) | Sanfilippo syndrome type B | Granting of the orphan status in the EU |
2017-08-29 | ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene | Abeona Therapeutics (USA - NY) Sermes Planificacion (Spain) | recessive dystrophic epidermolysis bullosa | Granting of the Breakthrough Therapy status |
2017-02-27 | 5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)pyridin-2-amine | Voisin Consulting (France) Piqur Therapeutics (Switzerland) | diffuse large B-cell lymphoma | Granting of the orphan status in the EU |
2017-02-27 | 26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound | Eirgen Pharma (UK) | Dravet syndrome | Granting of the orphan status in the EU |
2017-02-27 | alpha-tocopherol and ascorbic acid | Advanced Medical Projects (Spain) | fragile X syndrome | Granting of the orphan status in the EU |
2017-02-27 | cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl]acetate salt | Polyphor (Switzerland) | primary ciliary dyskinesia | Granting of the orphan status in the EU |
2017-06-19 | fenfluramine hydrochloride | Zogenix International (USA - CA) | Lennox-Gastaut syndrome | Granting of the orphan status in the US |
2017-02-27 | N-(4-(1-cyanocyclopentyl)phenyl)-2-(4-pyridinylmethyl)amino-3-pyridinecarboxamide methanesulfonate | Sirius Regulatory Consulting (UK) | gastric cancer | Granting of the orphan status in the EU |
2017-02-27 | recombinant human club cell 10 KDa protein | EUDRAC (Germany) | bronchiolitis obliterans syndrome | Granting of the orphan status in the EU |
2017-02-27 | 1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy- 2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropane-1-carboxamide and ivacaftor | Vertex Pharmaceuticals (USA - MA) | cystic fibrosis | Granting of the orphan status in the EU |
2017-02-27 | 505 amino acid protein, corresponding to amino acids 2-506 of the wild-type human histidyl-tRNA synthetase | Atyr Pharma (USA - CA) Voisin Consulting (France) | limb girdle muscular dystrophy 2B | Granting of the orphan status in the EU |
2017-02-27 | autologous T-cells transduced with lentiviral vector encoding an anti-SLAMF7 CD28/CD3-zeta chimeric antigen receptor | Dr. Michael Hudecek (Germany) | plasma cell myeloma/multiple myeloma | Granting of the orphan status in the EU |
2017-02-27 | ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene | Ser-mes Planificación SL (Spain) | epidermolysis bullosa | Granting of the orphan status in the EU |
2017-02-27 | humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 | Opsona Therapeutics (Ireland) |
|
Granting of the orphan status in the EU |
0000-00-00 | humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 | Opsona Therapeutics (Ireland) | myelodysplastic syndromes | Granting of the orphan status in the EU |
2017-02-27 | propranolol hydrochloride | Consejo Superior de Investigaciones Cientificas (CSIC) (Spain) | von Hippel-Lindau disease | Granting of the orphan status in the EU |
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