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Europe’s April 2014 Products Recommended For Orphan Drug Designation

April 15, 2014

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting April 8 – 9, 2014. The EMA COMP April 2014 Meeting Report on the review of applications for orphan designation is published April 14.

At this meeting, there are 12 positive opinions recommending the following medicines for designation as orphan medicinal products. COMP’s opinions are then 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 :

 Product Name Sponsor Company Indication
(5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-β-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9-tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one CellAct Pharma GmbH Biliary Tract Cancer
177Lu-tetraxetan-tetulomab Nordic Nanovector AS Follicular Lymphoma
Lutetium (177Lu) edotreotide ITG Isotope Technologies Garching GmbH Gastro-entero-pancreatic neuroendocrine tumors
Recombinant human alpha 1 chain homotrimer of type VII collagen Shire Pharmaceuticals (Ireland) Epidermolysis Bullosa
4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid Clanotech AB Prevention of scarring in post glaucoma filtration surgery
Adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1 Alan Boyd Consultants Ltd Choroideraemia
Aganirsen Gene Signal SAS Central retinal vein occlusion
Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene Cochamo Systems Ltd Mucopolysaccharidosis IIIA (Sanfilippo A syndrome) 
Autologous dendritic cells pulsed with RNA from glioma stem cells Epitarget AS Glioma
Isavuconazonium sulfate Astellas Pharma Europe Mucormycosis 
Paclitaxel-succinate-Arg-Arg-Leu-Ser-Tyr-Ser-Arg-Arg-Arg-Phe CLL Pharma Glioma
Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral,cationic lipid based gene transfer agent Imperial Innovations Limited Cystic Fibrosis

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

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