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Europe’s January 2014 Products Receiving Orphan Drug Designation

January 21, 2014

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) held a meeting January 7 – 8, 2014. The EMA COMP January 2014 Meeting Report on the review of applications for orphan designation is published January 16.

At this meeting, there are 15 positive opinions recommending orphan designation for the treatment of:

  Indication Sponsor Company Product Name
Cystic   Fibrosis Synovo   GmbH 1-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione
Cystic   Fibrosis Istituto   Europeo per la Ricerca sulla Fibrosi Cistica – ONLUS Cysteamine
Epidermolysis   Bullosa Prof.   Johann W. Bauer Diacerein
Prevention   of Delayed Graft Function after Solid Organ Transplantation Alexion   Europe SAS Eculizumab
Diagnosis   of Gastro-entero-pancreatic neuroendocrine tumors Advanced   Accelerator Applications SA Gallium   [Ga-68]-N-[(4,7,10-tricarboxymethyl-1,4,7,10tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(2-7)disulfide  
Congenital   Cytomegalovirus Infection Roche   Registration Limited Mixture of recombinant human   IgG1 monoclonal antibodies against human cytomegalovirus envelope   glycoproteins
Rett   Syndrome Neurolixis   UK Ltd 3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl)   amino]methyl}piperidin-1-yl]methanone
Gastro-entero-pancreatic   neuroendocrint tumors OctreoPharm   Sciences GmbH Ga-2,2′-(7-(4-((S)-1-((4S,7S,10S,13R,16S,19R)-4-((R)-1-amino-3-(4-hydroxyphenyl)-1-oxopropan-2-ylcarbamoyl)-10-(4-aminobutyl)-16-(4-((S)-2,6-dioxohexahydropyrimidine-4-carboxamido)benzyl)-7-((R)-1-hydroxyethyl)-6,9,12,15,18-pentaoxo-13-(4-ureidobenzyl)-1,2-dithia-5,8,11,14,17-pentaazacycloicosan-19-ylamino)-3-(4-chlorophenyl)-1-oxopropan-2-ylamino)-1-carboxy-4-oxobutyl)-1,4,7-triazonane-1,4-diyl)diacetic   acid
Duchenne   Muscular Dystrophy Gregory   Fryer Associates Ltd Asp-Arg-Val-Tyr-Ile-His-Pro
Glioma Diamond   BioPharm Limited Autologous dendritic cells   pulsed with tumour antigen-derived synthetic peptides (MAGE-1, HER-2, AIM-2,   TRP-2, gp-100, and interleukin-13 receptor alpha)
Optic Neuritis Bionure   Farma SL N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2-fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide
Familial   Chylomicronaemia Syndrome Isis   USA Ltd Phosphorothioate   oligonucleotide targeted to apolipoprotein C-III
Adrenoleuko-dystrophy Minoryx   Therapeutics S.L. Pioglitazone
Farber   Disease QOL   Therapeutics EU Ltd Recombinant human acid ceramidase
Polycythaemia Vera Novartis   Europharm Limited Ruxolitinib


Public summaries of the opinions will be available on the EMA website following adoption of the respective decisions on orphan designation by the European Commission.

Please Note: “Cambio de color a diferente pHs” by Rubashkyn (Own work) [CC-BY-SA-3.0] | Wikimedia Commons.

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