Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2014-10-17 | bortezomib | Millennium Pharmaceuticals (USA - MA), a Takeda company (Japan) | previously untreated patients with mantle cell lymphoma (MCL) |
Granting of a Market Authorisation in the US |
2015-02-16 | rufinamide | Eisai (Japan) |
|
Granting of a Market Authorisation in the US |
2015-03-31 | lanifibranor 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid | Inventiva (France) | systemic sclerosis | Granting of the orphan status in the US |
2014-11-19 | 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid | Inventiva (France) | idiopathic pulmonary fibrosis |
Granting of the orphan status in the EU |
2014-11-19 | 4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid | Coté Orphan Consulting UK Limited (UK) | cystic fibrosis |
Granting of the orphan status in the EU |
2015-06-01 | combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH | Apitope International (UK) | hemophilia A |
Granting of the orphan status in the EU |
2014-11-19 | donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment | Kiadis Pharma Netherlands (The Netherlands) | acute myeloid leukaemia | Granting of the orphan status in the EU |
2014-11-19 | imatinib | Numedicus Limited (UK) | acute respiratory distress syndrome |
Granting of the orphan status in the EU |
2018-10-18 | mexiletine hydrochloride | Lupin Europe (Germany) | symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders | Positive opinion for the granting of a Market Authorisation in the EU |
2014-11-19 | recombinant human pentraxin-2 | FGK Representative Service (Germany) | post-essential thrombocythaemia myelofibrosis |
Granting of the orphan status in the EU |
2014-11-19 | recombinant human pentraxin-2 | FGK Representative Service (Germany) | post-polycythaemia vera myelofibrosis |
Granting of the orphan status in the EU |
2014-11-19 | recombinant human pentraxin-2 | FGK Representative Service (Germany) | primary myelofibrosis |
Granting of the orphan status in the EU |
2014-11-19 | (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N\'-(pyrazin-2-yl)acrylohydrazide | Clinipace GmbH (Germany) | plasma cell myeloma |
Granting of the orphan status in the EU |
2014-11-19 | (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N\'-(pyrazin-2-yl)acrylohydrazide | Clinipace GmbH (Germany) | chronic lymphocytic leukaemia / small lymphocytic lymphoma |
Granting of the orphan status in the EU |
2014-11-19 | (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide | ChemoCentryx (USA - CA) | microscopic polyangiitis |
Granting of the orphan status in the EU |
2014-11-19 | (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide | ChemoCentryx (USA - CA) | granulomatosis with polyangiitis |
Granting of the orphan status in the EU |
2014-11-19 | adeno-associated viral vector serotype 8 containing the human MD1 gene | Genethon (France) | Duchenne muscular dystrophy |
Granting of the orphan status in the EU |
2015-01-13 | arimoclomol citrate | Orphazyme (Denmark) | Niemann-Pick disease, type C |
Granting of the orphan status in the US |
2014-11-19 | bazedoxifene acetate | Consejo Superior de Investigaciones Cientificas (CSIC) (Spain) | hereditary haemorrhagic telangiectasia |
Granting of the orphan status in the EU |
2015-05-20 | chloroquine | DualTpharma (The Netherlands) | glioma glioblastoma multiforme |
Granting of the orphan status in the US |
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