Date | Compound | Laboratory | Indication | Type of Information |
---|---|---|---|---|
2016-12-12 | metformin | Centro deInvestigación Biomédica en Red (CIBER) (Spain) | progressive myoclonic epilepsy type 2 (Lafora disease) |
Granting of the orphan status in the EU |
2016-12-12 | pegylated recombinant human interleukin-10 | Larode Ltd (UK) | pancreatic cancer |
Granting of the orphan status in the EU |
2016-12-12 | recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN3 gene | Ser-mes Planificación SL (Spain) | neuronal ceroid lipofuscinosis |
Granting of the orphan status in the EU |
2016-12-12 | udenafil | Mapi Ireland Limited | functional single ventricle congenital heart disease |
Granting of the orphan status in the EU |
2016-10-14 | (6aR,10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydrocannabinol-9-carboxylic acid | TMC Pharma Services (UK) | cystic fibrosis |
Granting of the orphan status in the EU |
2016-10-14 | non-covalent trimer of tumour necrosis factor fused to an antibody specific to the extra-domain Bof fibronectin in single-chain variable fragment format | Philogen (Italy) | soft tissue sarcoma |
Granting of the orphan status in the EU |
2016-07-25 | Philogen (Italy) | renal cell carcinoma and other CAIX-expressing tumours |
Granting of a patent | |
2016-10-14 | autologous mononuclear cells derived from human cord blood | BrainRepair UG (Germany) | periventricular leukomalacia |
Granting of the orphan status in the EU |
2016-10-14 | (E)-(6-((N-methyl-((3-methylbenzofuran-2-yl)methyl)amino)-3-oxoprop-1-en-1-yl)-2-oxo-3,4- dihydro-1,8-naphthyridin-1(2H)-yl)methyl phosphate, bis ethanolamine salt | Voisin Consulting (France) | osteomyelitis |
Granting of the orphan status in the EU |
2016-10-14 | human monoclonal IgG1 antibody against tissue factor pathway inhibitor | Pfizer (USA - NY) | hemophilia A |
Granting of the orphan status in the EU |
2016-10-14 | lutetium-177(3+), S2,S7-cyclo[N-{4,7,10-tricarboxymethyl-1,4,7,10-tetraaza-cyclododecan-1-ylacetyl}-4-chloro-L-phenylalanyl-D-cysteinyl-4-[(4S)-2,6-dioxo-1,3-diazinane-4-carboxamido]-Lphenylalanyl-4-(carbamoylamino)-D-phenylalanyl-L-lysyl-L-threonyl-L-cysteinyl-D-tyrosinamide] | Ipsen (France) | gastro-entero-pancreatic neuroendocrine tumours |
Granting of the orphan status in the EU |
2018-09-20 | mogamulizumab | Kyowa Hakko Kirin (Japan) | cutaneous T-cell lymphoma - mycosis fungoides - Sézary syndrome | Positive opinion for the granting of a Market Authorisation in the EU |
2016-10-14 | N-[(2S)-5-{[(1R, 2S)-2-(4-fluorophenyl)cyclopropyl]amino}-1-(4-methylpiperazin-1-yl)-1- oxopentan-2-yl]-4-(1H-1,2,3-triazol-1-yl)benzamide, bis-tosylate salt | Imago BioSciences (USA - CA) | myelofibrosis |
Granting of the orphan status in the EU |
2016-10-14 | radio-iodinated (131I) anti-CD45 murine monoclonal antibody | Wainwright Associates (UK) | hematopoietic stem cell transplantation |
Granting of the orphan status in the EU |
2016-10-14 | synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker | Ra Europe Limited (UK) | paroxysmal nocturnal haemoglobinuria |
Granting of the orphan status in the EU |
2016-10-14 | venetoclax | Abbvie (USA - IL) | multiple myeloma | Granting of the orphan status in the EU |
2016-10-14 | venetoclax | Abbvie (USA - IL) | diffuse large B-cell lymphoma | Granting of the orphan status in the EU |
2016-10-14 | xenon | Neuroprotexeon (UK) | ischaemia reperfusion injury associated with cardiac arrest |
Granting of the orphan status in the EU |
2016-10-14 | acebutolol hydrochloride | Therapicon (Italy) | Smith-Magenis syndrome |
Granting of the orphan status in the EU |
2016-10-14 | adeno-associated viral vector serotype 2/2 containing a gene encoding the channelrhodopsin-2 protein | Alacrita (UK) | retinitis pigmentosa |
Granting of the orphan status in the EU |
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