EMA Orphan Drug Designations – September 2014

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Find the FDA’s orphan designation and approvals for August here.

EMA – 5/09/2014
Drug: (S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile
Orphan Designation: pemphigus;
Sponsor: Almirall
EMA – 5/09/2014
Drug: (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide
Orphan Designation: Leigh syndrome
Sponsor: Khondrion
EMA – 5/09/2014
Drug: Cannabidiol
Orphan Designation: Dravet syndrome
Sponsor: GW Pharma
EMA – 5/09/2014
Drug: Cultured allogeneic corneal limbal stem cells
Orphan Designation: limbal stem cell deficiency
Sponsor: NHS National Services Scotland Trading as Scottish National Blood Transfusion Service
EMA – 5/09/2014
Drug: Cysteamine hydrochloride
Orphan Designation: cystinosis
Sponsor: Lucane Pharma
EMA – 5/09/2014
Drug: Immunoglobulin G1, anti-(human tumour-associated calcium signal transducer 2)(human-Mus musculus monoclonal hRS7 heavy chain), disulfide with human-Mus musculus monoclonal hRS7 κ-chain, dimer, hexakis(thioether) with (4S)-4-[[[[4-[[(2S)-2-(4-aminobutyl)-2-[[2-[2-[[26-[4-[[[[4-[(3-mercapto-2,5-dioxo-1-pyrrolidinyl)methyl]cyclohexyl]carbonyl]amino]methyl]-1H-1,2,3-triazol-1-yl]-3,6,9,12,15,18,21,24-octaoxahexacos-1-yl]amino]-2-oxoethoxy]acetyl]amino]-1-1-yl]-3,6,9,12,15,18,21,24-octaoxahexacos-1-yl]amino]-2-oxoethoxy]acetyl]amino]-1-pyrano[3′,4′:6,7]indolizino[1,2-b]quinoline-3,14(4H,12H)-dione
Orphan Designation: pancreatic cancer
Sponsor: Immunomedics
EMA – 5/09/2014
Drug: Osilodrostat
Orphan Designation: Cushing’s syndrome
Sponsor: Novartis
EMA – 5/09/2014
Drug: Oxalobacter formigenes strain HC-1
Orphan Designation:  short bowel syndrome
Sponsor: OxThera
EMA – 5/09/2014
Drug: Recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor
Orphan Designation: tenosynovial giant cell tumour, localised and diffused type
Sponsor: Novartis
EMA – 5/09/2014
Drug: (3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one
Orphan Designation: fragile X syndrome
Sponsor: Centre National de la Recherche Scientifique
EMA – 5/09/2014
Drug: Acamprosate calcium
Orphan Designation: fragile X syndrome
Sponsor: Real Regulatory Limited
EMA – 5/09/2014
Drug: Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
Orphan Designation: Crigler-Najjar syndrome
Sponsor: Genethon
EMA – 5/09/2014
Drug: Glucagon
Orphan Designation: congenital hyperinsulinism
Sponsor: S-Cubed
EMA – 5/09/2014
Drug: Nitric oxide
Orphan Designation: Cystic Fibrosis
Sponsor: PD Dr.med. Joachim Riethmüller
EMA – 5/09/2014
Drug: Pyridoxal 5′-phosphate
Orphan Designation: pyridoxamine 5′-phosphate oxidase deficienc
Sponsor: Great Ormond Street Hospital Foundation Trust
EMA – 5/09/2014
Drug: Raxibacumab
Orphan Designation: inhalation anthrax disease
Sponsor: GSK
EMA – 5/09/2014
Drug: Recombinant human bone morphogenetic protein 4
Orphan Designation: glioma
Sponsor: STEMGEN
EMA – 5/09/2014
Drug: Recombinant human insulin receptor monoclonal antibody-fused-α-L-iduronidase
Orphan Designation: mucopolysaccharidosis type I
Sponsor: Voisin Consulting
EMA – 5/09/2014
Drug: Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23
Orphan Designation: X-linked hypophosphataemia
Sponsor: NDA Group

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