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Europe’s September 2014 Products Recommended For Orphan Drug Designation

September 9, 2014

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting September 2 – 4, 2014.

At this meeting, there are 19 positive opinions recommending the following medicines for designation as orphan medicinal products. COMP’s opinions are forwarded to the European Commission (EC). The EC will then decide whether to grant an orphan designation for the medicines in question. Public summaries of the opinions will be available on the EMA website following adoption of the respective decisions on orphan designation by the EC.

EMA COMP September 2014 ODDs Recommended

Product Name Sponsor Company Indication
(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 Almirall S.A. Pemphigus
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride Khondrion BV Leigh Syndrome
Cannabidiol GW Pharma Ltd Dravet Syndrome
Cultured allogeneic corneal limbal stem cells NHS National Services Scotland Trading as Scottish National Blood Transfusion Service Limbal stem cell deficiency
Cysteamine hydrochloride Lucane Pharma SA Cystinosis
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-oxoethyl]amino]phenyl]methoxy]carbonyl]oxy]-4,11-diethyl-9-hydroxy-1H-pyrano[3′,4′:6,7]indolizino[1,2-b]quinoline-3,14(4H,12H)-dione Immunomedics GmbH Pancreatic Cancer
Osilodrostat Cushing’s Syndrome Novartis Europharm Limited
Oxalobacter formigenes strain HC-1 Short Bowel Syndrome OxThera AB
Recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor Tenosynovial Giant Cell Tumour Novartis Europharm Limited
(3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one Fragile X Syndrome Centre National de la Recherche Scientifique (CNRS)
Acamprosate Calcium Fagile X Syndrome Real Regulatory Limited
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene Crigler-Najjar Syndrome Généthon
Glucagon Congenital Hyperinsulinism S-Cubed Limited
Nitric Oxide Cystic Fibrosis Pd Dr. Med. Joachim Riethmuller
Pyridoxal 5′-phosphate Pyridoxamine 5′-phosphate oxidase deficiency Great Ormond Street Hospital Foundation Trust
Raxibacumab Inhalation Anthrax Disease GSK Trading Services Limited
Recombinant human bone morphogenetic protein 4 Glioma STEMGEN S.p.A.
Recombinant human insulin receptor monoclonal antibody-fused-α-L-iduronidase Mucopolysaccharidosis Type I Voisin Consulting S.A.R.L.
Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 X-Linked hypophosphatemia NDA Group AB


Please Note: “Erlenmeyer Flasks” From Argonne US National Lab  [Public domain in the US] | Wikimedia Commons.

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